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37 Study Matches

MT2013-31:Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis following Conditioning with Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG

To evaluate the ability to achieve high-level donor hematopoietic engraftment (defined as neutrophil recovery by Day +42 post-transplant and ≥ 80% donor cells on the myeloid fraction of peripheral blood at Day +100 post-transplant) using related and unrelated BM, PBSC, or UCB grafts following a reduced intensity conditioning regimen based on targeted-exposure busulfan, fludarabine +/- serotherapy in patients with inherited metabolic disorders and severe osteopetrosis.

Paul Orchard
All
up to 55 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT02171104
1406M51542
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Inclusion Criteria:

• 0 through 55 years of age
• Adequate graft available
• Adequate organ function
• Eligible Diseases:
• Mucopolysaccharidosis Disorders:
• MPS IH (Hurler syndrome)
• MPS II (Hunter syndrome) if the patient has no or minimal evidence of symptomatic neurologic disease but is expected to have a neurologic phenotype
• MPS VI (Maroteaux-Lamy syndrome)
• MPS VII (Sly syndrome)
• Glycoprotein Metabolic Disorders:
• Alpha mannosidosis
• Fucosidosis
• Aspartylglucosaminuria
• Sphingolipidoses and Recessive Leukodystrophies:
• Globoid cell leukodystrophy
• Metachromatic leukodystrophy
• Niemann-Pick B patients (sphingomyelin deficiency)
• Niemann-Pick C subtype 2
• Peroxisomal Disorders:
• Adrenoleukodystrophy with cerebral involvement
• Zellweger syndrome
• Neonatal Adrenoleukodystrophy
• Infantile Refsum disease
• Acyl-CoA-Oxidase Deficiency
• D-Bifunctional enzyme deficiency
• Multifunctional enzyme deficiency
• Alpha-methylacyl-CoA Racmase Deficiency (AMACRD)
• Mitochondrial Neurogastrointestingal Encephalopathy (MNGIE)
• Severe Osteopetrosis (OP)
• Hereditary Leukoencephalopathy with axonal spheroids (HDLS; CSF1R mutation)
• Other Inherited Metabolic Disorders (IMD): Patients will also be considered who have other life-threatening, rare lysosomal, peroxisomal or other similar inherited disorders characterized by white matter disease or other neurologic manifestations for which there is rationale that transplantation would be of benefit, such as certain patients with Wolman's disease, GM1 gangliosidosis, I-cell disease, Tay-Sachs disease, Sandhoff disease or others.
• Voluntary written consent
Exclusion Criteria:

• Pregnancy - menstruating females must have a negative serum or urine pregnancy test within 14 days of study treatment start
• Prior myeloablative chemotherapy exposure within 4 months of the start of conditioning on this protocol (patients excluded for this reason may be eligible for other institutional protocols)
• Uncontrolled bacterial, fungal or viral infections including HIV (including active infection with Aspergillus or other mold within 30 days)
Biological: Stem Cell Transplantation, Drug: IMD Preparative Regimen, Drug: Osteopetrosis Only Preparative Regimen, Drug: Osteopetrosis Haploidentical Only Preparative Regimen, Drug: cALD SR-A (Standard-Risk, Regimen A), Drug: cALD SR-B (Standard-Risk, Regimen B), Drug: cALD HR-D (High-Risk, Regimen C), Drug: cALD HR-D (High-Risk, Regimen D)
Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS, CSF1R Mutation), Inherited Metabolic Disorders
Clinics and Surgery Center (CSC), allogeneic hematopoietic cell transplantation, bone marrow transplantation, IMD, AMACRD, MNGIE, HDLS, OP, ALD
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An Observational Registry of Abatacept in Patients with Juvenile Idiopathic Arthritis (BMS Protocol IM101240)

The objective of this study is to create an international registry with long-term follow-up to characterize and evaluate the safety of abatacept in juvenile idiopathic arthritis (JIA). The primary objective of the JIA registry is to describe the long-term safety of abatacept treatment for JIA by quantifying the incidence rates of serious infections, autoimmune disorders, and malignancies.

Colleen Correll
All
up to 17 Years old
N/A
This study is NOT accepting healthy volunteers
NCT01357668
1403M48721
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For more information regarding BMS clinical trial participation, please visit www.BMSStudyConnect.com
Inclusion Criteria:

• Diagnosis of JIA (any subtype)
• Age < 18 years at the time of enrollment unless currently or previously enrolled in an abatacept clinical trial and received abatacept
• Receiving Abatacept at the time of enrollment as per treating physician's decision or received abatacept in a clinical trial
• Parent or legally acceptable representative willing to participate in the study and sign the informed consent
Exclusion Criteria:

• Pregnant or nursing female at the time of enrollment
• Prior malignancies if the patient has not been malignancy free for at least 5 years.
• Any serious acute or chronic medical condition other than JIA, including chronic infection, which would compromise the patient's ability to participate in the study
• Known poor compliance with clinic visits (based on physician judgment)
Juvenile Idiopathic Arthritis
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Observational Study of Pediatric Rheumatic Diseases: The CARRA Registry

The primary objective for this observational study is to collect general and medical data from children, adolescents, and young adults who had pediatric onset rheumatic disease. This data will be used to evaluate the long-term safety and efficacy of therapeutic agents used to treat these diseases. This information will allow investigators to accurately report and follow changes in current medication use patterns and compare these to proposed standards and current treatment recommendations. The use of a single registry will allow for more analysis of the different therapeutic agents by allowing them to be compared to each other.

Colleen Correll
All
up to 21 Years old
N/A
This study is NOT accepting healthy volunteers
NCT02418442
1506M74443
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Inclusion Criteria:

• Onset of rheumatic disease prior to age 16 years for JIA and onset prior to age 19 years for all other rheumatic diseases (see appendix A).
• Subject (and/or parent/legal guardian when required) is able to provide written informed consent and willing to comply with study procedures.
• Subject and/or parent/legal guardian is willing to be contacted in the future by study staff.
Exclusion Criteria:

• Greater than 21 years of age at the time of enrollment.
Rheumatic Joint Disease
Systemic Arthritis, Oligoarthritis, Polyarthritis (Rheumatoid Factor Negative), Polyarthritis (Rheumatoid Factor Positive), Psoriatic Arthritis, Enthesitis Related Arthritis (ERA), Undifferianted Arthritis, CARRA Consensus Treatment Plans
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Graded Motor Imagery for Women at Risk for Developing Type I CRPS following Distal Radius Fractures

Background: Distal radius fractures (DRF) account for nearly one-fifth of all fractures in older adults, and women experience them 5x as often as men. Most DRF occur with low impact injuries to the wrist with an outstretched hand, and are often managed via closed treatment and cast immobilization. Women sustaining a DRF are at risk for upper limb immobility, sensorimotor changes, edema and type I complex regional pain syndrome (CRPS). Since CRPS onset is likely influenced by alterations in the brain’s somatosensory region, a rehabilitation intervention, Graded Motor Imagery (GMI), aims to restore cortical representation, including sensory and motor function, of the affected limb. To date, there are no studies on the use of GMI in reducing risk of or preventing the onset of type I CRPS in women with DRF treated with cast immobilization. Due to a higher likelihood of women with this injury developing type I CRPS, it is important to early intervention is needed. Methods/Design: This article describes a six-week randomized comparative effectiveness trial, where the outcomes of a modified GMI program (mGMI) + standard of care (SOC) group (n=33) are compared to a SOC only control group (n=33). Immediately following cast immobilization, both groups participate in four 1-hour clinic-based sessions, and a home program for 10 minutes three times daily until cast removal. Blinded assessments occur within 1 week of cast immobilization (baseline), at three weeks post cast immbolization, cast removal, and at three months post cast removal. The primary outcomes are patient reported wrist/hand function and symptomology on the Patient Rated Wristand Hand Evaluation, McGill Pain Questionnaire, and Budapest CRPS Criteria. The secondary outcomes are grip strength, active range of motion as per goniometry, circumferential edema measurements, and joint position sense. Discussion: This study will investigate the early effects of mGMI + SOC hand therapy compared to SOC alone. We intend to investigate whether an intervention, specifically mGMI, used to treat preexisiting pain and motor dysfunction might also be used to mitigate these problems prior to their onset. If positive effects are observed, mGMI + SOC may be considered for incorporation into early rehabilitation program.

Corey McGee, PhD, MS, OTR/L, CHT
Female
55 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT02957240
1701M03721
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Inclusion Criteria:

• Women 55 years or older who have received closed treatment of distal radius fractures
Exclusion Criteria:

• Central nervous system disorders (e.g., Brain injury, Spinal Cord Injury, Parkinson's, Multiple Sclerosis)
• Surgical fixation of fracture
• Non english speaking
• Concomitant ipsilateral injuries (i..e., BBFF)
• Other injuries to the affected limb interfering with baseline affected limb function
• Cognitive disorders which would preclude from following the testing commands and home program participation
• Conditions of the contralateral upper limb which would result in painful and markedly limited active hand, wrist and forearm motion as this may impact the brain's ability to perceive safe and proficient movement during mirror therapy.
• Visual impairments resulting in the inability to participate in GMI components
Behavioral: Standard Care, Behavioral: Motor Representation Techniques
Musculoskeletal Pain, Fractures, Closed, Distal Radius Fracture, Complex Regional Pain Syndromes
Forearm [A01.378.800.585], Radius [A02.835.232.087.090.700], Motor Skills [F02.808.260], Task Performance and Analysis [F02.808.600], Casts, Surgical [E07.858.442.660.430.500], Splints [E07.858.690.725.430.750], motor representation techniques, mirror therapy, Women [M01.975], Clinics and Surgery Center (CSC)
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Use of Entresto (sacubitril/valsartan) for the treatment of peripheral arterial disease

The primary objective of this study is to assess the effects of 12 weeks of Entresto on the functional clinical parameters maximal walking duration, pain free walking duration and 6 minute walk test of patients with PAD. In addition, to determine the effects of Entresto on peak VO2, cardiac output and systemic vascular resistance.

Otto Sanchez
All
18 Years to 80 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT02636283
1702M08001
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Inclusion Criteria:

• Subject with symptoms of intermittent claudication, such as exercise-induced pain, cramps, fatigue, or other equivalent discomfort, involving large muscle groups of the leg(s) (calf, thigh, buttocks), relieved by rest.
• Ankle-brachial index ≤ 0.90 acquired according to the American Heart Association guidelines.
• Highest ankle pressure reduced by at least 25 mm Hg after exercise compared to resting pressure (or loss of previously present Doppler signal for both the posterior tibial and anterior tibial arteries immediately after exercise if arteries were incompressible).
• Patients on medical treatment for PAD without significant improvement in intermittent claudication within the last 6 months.
Exclusion Criteria:

• Age < 18 and > 80 years.
• Patients with physician diagnosed chronic kidney disease or heart failure stage II or IV or unstable angina.
• Echocardiographic evidence of cardiomyopathies and pulmonary hypertension.
• Patients that have received cancer treatment within the last year (except skin cancer).
• Severe limitations in mobility due to osteomuscular disorders present at time of interview.
• Dementia or other mental disorders that prevent patients from following a research protocol present at time of interview
• Patients engaged in an exercise rehabilitation program within the past 6 months.
• Patients schedule to undergo an arterial revascularization procedure during the study or have undergone one within the past 6 months.
• Inconsistent maximal walking distance on the treadmill test.
Drug: Entresto, Drug: Placebo group
Peripheral Arterial Disease
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COG APEC1621F - NCI-COG PEDIATRIC MATCH (MOLECULAR ANALYSIS FOR THERAPY CHOICE)- PHASE 2 SUBPROTOCOL OF ENSARTINIB IN PATIENTS WITH TUMORS HARBORING ALK OR ROS1 GENOMIC ALTERATIONS

The NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) study, referred to as Pediatric MATCH, will match targeted agents with specific molecular changes identified using genomic sequencing technologies in refractory/recurrent tumors from children and adolescents with cancer. The NCI-COG Pediatric MATCH study (APEC1621) is considered one study under one IND consisting of a Master Version Control protocol, screening protocol component and multiple subprotocol components, with each component being contained in its own separate document. Each “component” consists of the protocol document and its associated informed consent document. The Master Version Control Protocol is the overarching administrative protocol that will keep an up-to-date record of the current version of the screening protocol component and each subprotocol component of Pediatric MATCH. Since each subprotocol component operates independently from the other subprotocol components contained in Pediatric MATCH, each has its own version date. Likewise, the screening protocol component has its own version date since changes in any of the subprotocols may not require changes in the screening protocol.

Emily Greengard
All
12 Months to 21 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03213652
STUDY00001752
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Inclusion Criteria:

• Patient must have enrolled onto APEC1621SC and must have been given a treatment assignment to Molecular Analysis for Therapy Choice (MATCH) to APEC1621F based on the presence of an actionable mutation
• Patients must have a body surface area >= 0.5 m^2 at enrollment
• Patients must have radiographically measurable disease at the time of study enrollment. Patients with neuroblastoma who do not have measurable disease but have iobenguane (MIBG) positive (+) evaluable disease are eligible; measurable disease in patients with CNS involvement is defined as any lesion that is at minimum 10 mm in one dimension on a standard magnetic resonance imaging (MRI) or computed tomography (CT)
• Note: The following do not qualify as measurable disease:
• Malignant fluid collections (e.g., ascites, pleural effusions)
• Bone marrow infiltration except that detected by MIBG scan for neuroblastoma
• Lesions only detected by nuclear medicine studies (e.g., bone, gallium or positron emission tomography [PET] scans) except as noted for neuroblastoma
• Elevated tumor markers in plasma or cerebrospinal fluid (CSF)
• Previously radiated lesions that have not demonstrated clear progression post radiation
• Leptomeningeal lesions that do not meet the measurement requirements for Response Evaluation Criteria in Solid Tumors (RECIST) 1.1
• Karnofsky >= 50% for patients > 16 years of age and Lansky >= 50 for patients =< 16 years of age
• Note: Neurologic deficits in patients with CNS tumors must have been relatively stable for at least 7 days prior to study enrollment; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
• Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment; if after the required timeframe, the numerical eligibility criteria are met, e.g. blood count criteria, the patient is considered to have recovered adequately
• Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive: >= 21 days after the last dose of cytotoxic or myelosuppressive chemotherapy (42 days if prior nitrosourea)
• Anti-cancer agents not known to be myelosuppressive (e.g. not associated with reduced platelet or absolute neutrophil counts [ANC] counts): >= 7 days after the last dose of agent
• Antibodies: >= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =< 1
• Corticosteroids: if used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Hematopoietic growth factors: >= 14 days after the last dose of a long-acting growth factor (e.g. pegfilgrastim) or 7 days for short-acting growth factor; for growth factors that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur; the duration of this interval must be discussed with the study chair and the study-assigned research coordinator
• Interleukins, interferons and cytokines (other than hematopoietic growth factors): >= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors)
• Stem cell Infusions (with or without total body irradiation [TBI]):
• Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor lymphocyte infusion (DLI) or boost infusion: >= 84 days after infusion and no evidence of graft versus host disease (GVHD)
• Autologous stem cell infusion including boost infusion: >= 42 days
• Cellular therapy: >= 42 days after the completion of any type of cellular therapy (e.g. modified T cells, natural killer [NK] cells, dendritic cells, etc.)
• Radiation therapy (XRT)/external beam irradiation including protons: >= 14 days after local XRT; >= 150 days after TBI, craniospinal XRT or if radiation to >= 50% of the pelvis; >= 42 days if other substantial none marrow (BM) radiation
• Note: Radiation may not be delivered to "measurable disease" tumor site(s) being used to follow response to subprotocol treatment
• Radiopharmaceutical therapy (e.g., radiolabeled antibody, 131I-MIBG): >= 42 days after systemically administered radiopharmaceutical therapy
• Patients must not have received prior exposure to ensartinib; prior treatment with other ALK inhibitors is permitted given that at least 5 half-lives or 21 days have elapsed since therapy discontinuation, whichever is greater
• For patients with solid tumors without known bone marrow involvement:
• Peripheral absolute neutrophil count (ANC) >= 1000/mm^3
• Platelet count >= 100,000/mm^3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment)
• Patients with known bone marrow metastatic disease will be eligible for study provided they meet the blood counts (may receive transfusions provided they are not known to be refractory to red cell or platelet transfusions); these patients will not be evaluable for hematologic toxicity
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 ml/min/1.73 m^2 (within 7 days prior to enrollment) or a serum creatinine based on age/gender as follows (within 7 days prior to enrollment):
• Age 1 to < 2 years: maximum serum creatinine 0.6 mg/dL for male and 0.6 mg/dL for female
• Age 2 to < 6 years: maximum serum creatinine 0.8 mg/dL for male and 0.8 mg/dL for female
• Age 6 to < 10 years: maximum serum creatinine 1 mg/dL for male and 1 mg/dL for female
• Age 10 to < 13 years: maximum serum creatinine 1.2 mg/dL for male and 1.2 mg/dL for female
• Age 13 to < 16 years: maximum serum creatinine 1.5 mg/dL for male and 1.4 mg/dL for female
• Age >= 16 years: maximum serum creatinine 1.7 mg/dL for male and 1.4 mg/dL for female
• Bilirubin (sum of conjugated + unconjugated) =< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment)
• Serum glutamic pyruvic transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135 U/L (within 7 days prior to enrollment) (for the purpose of this study, the ULN for SGPT is 45 U/L)
• Serum albumin >= 2 g/dL (within 7 days prior to enrollment)
• Patients must be able to swallow intact capsules
• All patients and/or their parents or legally authorized representatives must sign a written informed consent; assent, when appropriate, will be obtained according to institutional guidelines
Exclusion Criteria:

• Pregnant or breast-feeding women will not be entered on this study because there is currently no available information regarding human fetal or teratogenic toxicities; pregnancy tests must be obtained in girls who are post-menarchal; males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method for the duration of study treatment and for one week after the last dose of ensartinib
• Concomitant medications
• Corticosteroids: patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible; if used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Investigational drugs: patients who are currently receiving another investigational drug are not eligible
• Anti-cancer agents: patients who are currently receiving other anti-cancer agents are not eligible
• Anti-GVHD agents post-transplant: patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible for this trial
• CYP3A4 agents: patients who are currently receiving drugs that are strong inducers or strong inhibitors of CYP3A4 are not eligible; strong inducers or inhibitors of CYP3A4 should be avoided from 14 days prior to enrollment to the end of the study
• Note: CYP3A4 inducing anti-epileptic drugs and dexamethasone for CNS tumors or metastases, on a stable dose, are allowed
• Patients who have an uncontrolled infection are not eligible
• Patients who have received a prior solid organ transplantation are not eligible
• Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible
Drug: Ensartinib, Other: Laboratory Biomarker Analysis, Other: Pharmacological Study
Malignant Solid Neoplasm, Recurrent Ependymoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Glioma, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Recurrent WHO Grade 2 Glioma, Refractory Ependymoma, Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Germ Cell Tumor, Refractory Malignant Glioma, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Wilms Tumor
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Larotrectinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With NTRK Fusions (A Pediatric MATCH Treatment Trial)

The NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) study, referred to as Pediatric MATCH, will match targeted agents with specific molecular changes identified using genomic sequencing technologies in refractory/recurrent tumors from children and adolescents with cancer. The NCI-COG Pediatric MATCH study (APEC1621) is considered one study under one IND consisting of a Master Version Control protocol, screening protocol component and multiple subprotocol components, with each component being contained in its own separate document. Each “component” consists of the protocol document and its associated informed consent document. The Master Version Control Protocol is the overarching administrative protocol that will keep an up-to-date record of the current version of the screening protocol component and each subprotocol component of Pediatric MATCH. Since each subprotocol component operates independently from the other subprotocol components contained in Pediatric MATCH, each has its own version date. Likewise, the screening protocol component has its own version date since changes in any of the subprotocols may not require changes in the screening protocol.

Emily Greengard
All
12 Months to 21 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03213704
STUDY00001752
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Inclusion Criteria:

• APEC1621SC: Patient must have enrolled onto APEC1621SC and must have been given a treatment assignment to Molecular Analysis for Therapy Choice (MATCH) to APEC1621A based on the presence of an actionable mutation as defined in APEC1621SC
• Patients must have radiographically measurable disease at the time of study enrollment; patients with neuroblastoma who do not have measurable disease but have iobenguane (MIBG)+ evaluable disease are eligible; measurable disease in patients with CNS involvement is defined as any lesion that is at minimum 10 mm in one dimension on standard magnetic resonance imaging (MRI) or computed tomography (CT); Note: The following do not qualify as measurable disease:
• Malignant fluid collections (e.g., ascites, pleural effusions)
• Bone marrow infiltration except that detected by MIBG scan for neuroblastoma
• Lesions only detected by nuclear medicine studies (e.g., bone, gallium or positron emission tomography [PET] scans) except as noted for neuroblastoma
• Elevated tumor markers in plasma or cerebrospinal fluid (CSF)
• Previously radiated lesions that have not demonstrated clear progression post radiation
• Leptomeningeal lesions that do not meet the measurement requirements for Response Evaluation Criteria in Solid Tumors (RECIST) 1.1
• Karnofsky >= 50% for patients > 16 years of age and Lansky >= 50 for patients =< 16 years of age; Note: neurologic deficits in patients with CNS tumors must have been relatively stable for at least 7 days prior to study enrollment; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
• Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment; if after the required time frame, the numerical eligibility criteria are met, e.g. blood count criteria, the patient is considered to have recovered adequately
• Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive; for agents not listed, the duration of this interval must be discussed with the study chair and the study-assigned research coordinator prior to enrollment
• >= 21 days after the last dose of cytotoxic or myelosuppressive chemotherapy (42 days if prior nitrosourea)
• Anti-cancer agents not known to be myelosuppressive (e.g. not associated with reduced platelet or absolute neutrophil [ANC] counts): >= 7 days after the last dose of agent; for agents not listed, the duration of this interval must be discussed with the study chair and the study-assigned research coordinator prior to enrollment
• Antibodies: >= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =< 1
• Corticosteroids: if used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Hematopoietic growth factors: >= 14 days after the last dose of a long-acting growth factor (e.g. pegfilgrastim) or 7 days for short-acting growth factor; for growth factors that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur; the duration of this interval must be discussed with the study chair and the study-assigned research coordinator
• Interleukins, interferons and cytokines (other than hematopoietic growth factors): >= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors)
• Stem cell Infusions (with or without total body irradiation [TBI]):
• Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor leukocyte infusion (DLI) or boost infusion: >= 84 days after infusion and no evidence of graft versus host disease (GVHD)
• Autologous stem cell infusion including boost infusion: >= 42 days
• Cellular therapy: >= 42 days after the completion of any type of cellular therapy (e.g. modified T cells, NK cells, dendritic cells, etc.)
• Radiation therapy (XRT)/external beam irradiation including protons: >= 14 days after local XRT; >= 150 days after TBI, craniospinal XRT or if radiation to >= 50% of the pelvis; >= 42 days if other substantial brain metastases (BM) radiation; Note: radiation may not be delivered to "measurable disease" tumor site(s) being used to follow response to subprotocol treatment
• Radiopharmaceutical therapy (e.g., radiolabeled antibody, 131I-MIBG): >= 42 days after systemically administered radiopharmaceutical therapy
• Patients must not have received prior exposure to other NTRK inhibitors including but not limited to LOXO-101 (larotrectinib), entrectinib (RXDX-101), DS6051, PLX7486
• For patients with solid tumors without known bone marrow involvement:
• Peripheral absolute neutrophil count (ANC) >= 1000/mm^3
• Platelet count >= 100,000/mm^3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment)
• Patients with known bone marrow metastatic disease will be eligible for study provided they meet the blood counts (may receive transfusions provided they are not known to be refractory to red cell or platelet transfusions); these patients will not be evaluable for hematologic toxicity
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 ml/min/1.73 m^2 or
• A serum creatinine based on age/gender as follows:
• Age: 1 to < 2 years; maximum serum creatinine (mg/dL); male: 0.6 female: 0.6
• Age: 2 to < 6 years; maximum serum creatinine (mg/dL); male: 0.8 female: 0.8
• Age: 6 to < 10 years; maximum serum creatinine (mg/dL); male: 1 female: 1
• Age: 10 to < 13 years; maximum serum creatinine (mg/dL); male: 1.2 female: 1.2
• Age: 13 to < 16 years; maximum serum creatinine (mg/dL); male: 1.5 female: 1.4
• Age: >= 16 years; maximum serum creatinine (mg/dL); male: 1.7 female: 1.4
• Bilirubin (sum of conjugated + unconjugated) =< 1.5 x upper limit of normal (ULN) for age
• Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135 U/L; (for the purpose of this study, the ULN for SGPT is 45 U/L)
• Serum albumin >= 2 g/dL
• Patients with seizure disorder may be enrolled if on anti-convulsants and well controlled
• Nervous system disorders (Common Terminology Criteria for Adverse Events [CTCAE] version [v] 5.0) resulting from prior therapy must be =< grade 2, with the exception of decreased tendon reflect (DTR); any grade of DTR is eligible
• All patients and/or their parents or legally authorized representatives must sign a written informed consent; assent, when appropriate, will be obtained according to institutional guidelines
Exclusion Criteria:

• Pregnant or breast-feeding women will not be entered on this study due to risks of fetal and teratogenic adverse events as seen in animal/human studies; pregnancy tests must be obtained in girls who are post-menarchal; males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method for the duration of study treatment
• Patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible; if used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Patients who are currently receiving another investigational drug are not eligible
• Patients who are currently receiving other anti-cancer agents are not eligible
• Patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease (GVHD) post bone marrow transplant are not eligible for this trial
• Patients who are currently receiving drugs that are strong inducers or inhibitors of CYP3A4 are not eligible; strong inducers or inhibitors of CYP3A4 should be avoided from 14 days prior to enrollment to the end of the study; Note: CYP3A4 inducing anti-epileptic drugs and dexamethasone for CNS tumors or metastases, on a stable dose, are allowed
• Patients who have received prior therapy with a specific inhibitor of TRK (including but not limited to entrectinib [RXDX-101], DS-6051b, and PLX7486) are not eligible
• Patients who have an uncontrolled infection are not eligible
• Patients who have received a prior solid organ transplantation are not eligible
• Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible
Drug: Larotrectinib Sulfate
Advanced Malignant Solid Neoplasm, Recurrent Ependymoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Glioma, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Glioma, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Rhabdoid Tumor, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Refractory Ependymoma, Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Refractory Glioma, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Germ Cell Tumor, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Primary Central Nervous System Neoplasm, Refractory Rhabdoid Tumor, Refractory Rhabdomyosarcoma, Wilms Tumor
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COG APEC1621SC - NCI-COG PEDIATRIC MATCH (MOLECULAR ANALYSIS FOR THERAPY CHOICE) SCREENING PROTOCOL

The NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) study, referred to as Pediatric MATCH, will match targeted agents with specific molecular changes identified using genomic sequencing technologies in refractory/recurrent tumors from children and adolescents with cancer. The NCI-COG Pediatric MATCH study (APEC1621) is considered one study under one IND consisting of a Master Version Control protocol, screening protocol component and multiple subprotocol components, with each component being contained in its own separate document. Each “component” consists of the protocol document and its associated informed consent document. The Master Version Control Protocol is the overarching administrative protocol that will keep an up-to-date record of the current version of the screening protocol component and each subprotocol component of Pediatric MATCH. Since each subprotocol component operates independently from the other subprotocol components contained in Pediatric MATCH, each has its own version date. Likewise, the screening protocol component has its own version date since changes in any of the subprotocols may not require changes in the screening protocol.

Emily Greengard
All
12 Months to 21 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03155620
STUDY00001752
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Inclusion Criteria:

• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Patients must be >= 12 months and =< 21 years of age at the time of study enrollment
• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Patients with recurrent or refractory solid tumors, including non-Hodgkin lymphomas, histiocytoses (e.g. langerhans cell histiocytosis [LCH], juvenile xanthogranuloma [JXG], histiocytic sarcoma), and central nervous system (CNS) tumors are eligible; patients must have had histologic verification of malignancy at original diagnosis or relapse except in patients with intrinsic brain stem tumors, optic pathway gliomas, or patients with pineal tumors and elevations of cerebrospinal fluid (CSF) or serum tumor markers including alpha-fetoprotein or beta-human chorionic gonadotropin (HCG); in cases where patient enrolls prior to histologic confirmation of recurrent disease, patient is ineligible and should be withdrawn from study if histology fails to confirm recurrence; please note: Patients with Hodgkin lymphoma and plexiform neurofibroma are not eligible
• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Tumor Testing Requirement: Tumor sample availability requirement for stage 1 of Pediatric MATCH (patients enrolled from start of study in July 2017 through 12/31/21); Patients must have an formalin-fixed paraffin-embedded (FFPE) tumor sample available for MATCH study testing from a biopsy or surgery that was performed at any point after initial tumor recurrence/progression, or be planned to have a procedure to obtain such a sample that is considered to be of potential benefit by the treating clinicians; a tumor sample from a clinically performed diagnostic (pre-treatment) biopsy will be acceptable for enrollment onto Pediatric MATCH only for children with high-grade gliomas of the brainstem (diffuse intrinsic pontine gliomas) or thalamus
• Please note: Samples that have been decalcified using standardly utilized acid-based decalcification methods are not generally suitable for MATCH study testing; the nucleic acids will have been degraded in the decalcification process
• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Tumor molecular profiling report availability requirement for Stage 2 of Pediatric MATCH (patients enrolled starting 2022): In stage 2 of the study, no tumor samples will be submitted for centralized clinical tumor profiling; instead, a tumor molecular profiling report from a College of American Pathologists (CAP)/ Clinical Laboratory Improvements Amendments (CLIA)-approved testing laboratory must be submitted for review by the Molecular Review Committee (MRC)
• This molecular profiling must have been performed on a tumor sample that was obtained at any point after initial tumor recurrence/progression and must be accompanied by a pathology report for the same tumor specimen; a molecular profiling report for a diagnostic (pre-treatment) tumor sample will be acceptable for enrollment onto Pediatric MATCH only for children with high-grade gliomas of the brainstem (diffuse intrinsic pontine gliomas) or thalamus. In the event that molecular profiling reports are available from multiple timepoints, the most recent report should be prioritized for study submission
• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Karnofsky >= 50% for patients > 16 years of age and Lansky >= 50 for patients =< 16 years of age); note: neurologic deficits in patients with central nervous system (CNS) tumors must have been stable for at least 7 days prior to study enrollment; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Patients must have radiographically measurable disease; measurable disease based on imaging obtained less than or equal to 56 days prior to enrollment; patients with neuroblastoma who do not have measurable disease but have metaiodobenzylguanidine (MIBG) positive (+) evaluable disease are eligible; measurable disease in patients with CNS involvement is defined as any lesion that is at minimum 10 mm in one dimension on standard magnetic resonance imaging (MRI) or computed tomography (CT)
• Note: The following do not qualify as measurable disease:
• Malignant fluid collections (e.g., ascites, pleural effusions)
• Bone marrow infiltration except that detected by MIBG scan for neuroblastoma
• Lesions only detected by nuclear medicine studies (e.g., bone, gallium or positron emission tomography [PET] scans) except as noted for neuroblastoma
• Elevated tumor markers in plasma or CSF
• Previously radiated lesions that have not demonstrated clear progression post radiation
• Leptomeningeal lesions that do not meet the measurement requirements for Response Evaluation Criteria in Solid Tumors (RECIST) 1.1
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: NOTE: patient does not need to meet all subprotocol criteria at time of enrollment onto the APEC1621SC screening protocol, but will need to meet all criteria prior to enrollment on any assigned treatment subprotocol. Patients must be enrolled onto a subprotocol within 2 weeks (14 days) of treatment assignment
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Karnofsky >= 50% for patients > 16 years of age and Lansky >= 50 for patients =< 16 years of age); Note: neurologic deficits in patients with CNS tumors must have been stable for at least 7 days prior to study enrollment; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: At the time of treatment with subprotocol specified therapy, the patients must have radiographically measurable disease; patients with neuroblastoma who do not have measurable disease but have MIBG+ evaluable are eligible; measurable disease in patients with CNS involvement is defined as any lesion that is at minimum 10 mm in one dimension on standard MRI or CT
• Note: The following do not qualify as measurable disease:
• Malignant fluid collections (e.g., ascites, pleural effusions)
• Bone marrow infiltration except that detected by MIBG scan for neuroblastoma
• Lesions only detected by nuclear medicine studies (e.g., bone, gallium or positron emission tomography [PET] scans) except as noted for neuroblastoma
• Elevated tumor markers in plasma or CSF
• Previously radiated lesions that have not demonstrated clear progression post radiation
• Leptomeningeal lesions that do not meet the measurement requirements for RECIST 1.1
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: At the time of enrollment onto a subprotocol, the following general criteria for initiation of therapy will be required:
• Patients must have fully recovered from the acute toxic effects of all prior anticancer therapy and must meet the following minimum duration from prior anticancer directed therapy prior to enrollment to the subprotocol; if after the required timeframe, the numerical eligibility criteria are met, e.g. blood count criteria, the patient is considered to have recovered adequately
• Cytotoxic chemotherapy or other anticancer agents known to be myelosuppressive: for agents not listed, the duration of this interval must be discussed with the study chair and the study-assigned research coordinator prior to enrollment >= 21 days after the last dose of cytotoxic or myelosuppressive chemotherapy (42 days if prior nitrosourea)
• Anticancer agents not known to be myelosuppressive (e.g. not associated with reduced platelet or absolute neutrophil counts [ANC]): >= 7 days after the last dose of agent; for agents not listed, the duration of this interval must be discussed with the study chair and the study-assigned research coordinator prior to enrollment
• Antibodies: >= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =< 1
• Corticosteroids: If used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Hematopoietic growth factors: >= 14 days after the last dose of a long-acting growth factor (e.g. Neulasta) or 7 days for short-acting growth factor; for agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur; the duration of this interval must be discussed with the study chair and the study-assigned research coordinator
• Interleukins, interferons and cytokines (other than hematopoietic growth factors): >= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors)
• Stem cell infusions (with or without total-body irradiation [TBI]):
• Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor lymphocyte infusion (DLI) or boost infusion: >= 84 days after infusion and no evidence of graft versus host disease (GVHD)
• Autologous stem cell infusion including boost infusion: >= 42 days
• Cellular therapy: >= 42 days after the completion of any type of cellular therapy (e.g. modified T cells, natural killer (NK) cells, dendritic cells, etc.)
• X-ray therapy (XRT)/External Beam Irradiation including Protons: >= 14 days after local XRT; >= 150 days after TBI, craniospinal XRT or if radiation to >= 50% of the pelvis; >= 42 days if other substantial bone marrow (BM) radiation; note: radiation may not be delivered to "measurable disease" tumor site(s) being used to follow response to subprotocol treatment
• Radiopharmaceutical therapy (e.g., radiolabeled antibody, 131I-MIBG): >= 42 days after systemically administered radiopharmaceutical therapy
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: For patients with solid tumors without known bone marrow involvement:
• Peripheral absolute neutrophil count (ANC) >= 1000/mm^3
• Platelet count >= 100,000/mm^3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment)
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Patients with known bone marrow metastatic disease will be eligible for study provided they meet the blood counts (may receive transfusions provided they are not known to be refractory to red cell or platelet transfusions); these patients will not be evaluable for hematologic toxicity
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 ml/min/1.73 m^2 or a serum creatinine based on age/gender as follows:
• Age: 1 to < 2 years; maximum serum creatinine (mg/dL): male 0.6; female 0.6
• Age: 2 to < 6 years; maximum serum creatinine (mg/dL): male 0.8; female 0.8
• Age: 6 to < 10 years; maximum serum creatinine (mg/dL): male 1; female 1
• Age: 10 to < 13 years; maximum serum creatinine (mg/dL): male 1.2; female 1.2
• Age: 13 to < 16 years; maximum serum creatinine (mg/dL): male 1.5; female 1.4
• Age: >= 16 years; maximum serum creatinine (mg/dL): male 1.7; female 1.4
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Bilirubin (sum of conjugated + unconjugated) =< 1.5 x upper limit of normal (ULN) for age
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Serum glutamate pyruvate transaminase (SGPT) (alanine transferase [ALT]) =< 135 U/L (for the purpose of this study, the ULN for SGPT is 45 U/L)
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Patients must be able to swallow intact capsules/tablets, unless otherwise specified in the subprotocol to which they are assigned
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Agent specific limitations on prior therapy will be included with specific treatment subprotocols
Exclusion Criteria:

• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Pregnant or breast-feeding women will not be entered on this study due to risks of fetal and teratogenic adverse events as seen in animal/human studies, or because there is currently no available information regarding human fetal or teratogenic toxicities; pregnancy tests must be obtained in females who are post-menarchal; males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method
• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Concomitant medications
• Corticosteroids: at the time of consent and enrollment to regimen specific subprotocols, patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment to the subprotocol will not be eligible; if used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Investigational drugs: patients must meet criteria for prior therapy at the time of consent and enrollment to a subprotocol; other investigational agents may not be administered to patients while they are receiving study drug as part of a subprotocol
• Anticancer agents: patients must meet criteria for prior therapy at the time of consent and enrollment to a subprotocol; other investigational agents may not be administered to patients while they are receiving study drug as part of a subprotocol
• Anti-GVHD agents post-transplant: patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible
• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Patients who have an uncontrolled infection are not eligible
• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Patients who have had a prior solid organ transplant are not eligible
• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Additional agent specific criteria will be included with specific treatment subprotocols
Procedure: Biopsy, Procedure: Biospecimen Collection, Drug: Ensartinib, Drug: Erdafitinib, Other: Laboratory Biomarker Analysis, Drug: Larotrectinib Sulfate, Procedure: Mutation Carrier Screening, Drug: Olaparib, Drug: Palbociclib, Other: Pharmacological Study, Drug: Samotolisib, Drug: Selpercatinib, Drug: Selumetinib Sulfate, Drug: Tazemetostat, Drug: Tipifarnib, Drug: Ulixertinib, Drug: Vemurafenib
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Non-Hodgkin Lymphoma, Ann Arbor Stage IV Non-Hodgkin Lymphoma, Histiocytic Sarcoma, Juvenile Xanthogranuloma, Langerhans Cell Histiocytosis, Malignant Glioma, Recurrent Childhood Rhabdomyosarcoma, Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Glioma, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Peripheral Primitive Neuroectodermal Tumor, Recurrent Primary Central Nervous System Neoplasm, Recurrent Rhabdoid Tumor, Recurrent Soft Tissue Sarcoma, Refractory Ewing Sarcoma, Refractory Glioma, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Germ Cell Tumor, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Peripheral Primitive Neuroectodermal Tumor, Refractory Primary Central Nervous System Neoplasm, Refractory Rhabdoid Tumor, Refractory Rhabdomyosarcoma, Rhabdoid Tumor, Stage III Osteosarcoma AJCC v7, Stage III Soft Tissue Sarcoma AJCC v7, Stage IV Osteosarcoma AJCC v7, Stage IV Soft Tissue Sarcoma AJCC v7, Stage IVA Osteosarcoma AJCC v7, Stage IVB Osteosarcoma AJCC v7, Wilms Tumor
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Samotolisib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With TSC or PI3K/MTOR Mutations (A Pediatric MATCH Treatment Trial)

The NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) study, referred to as Pediatric MATCH, will match targeted agents with specific molecular changes identified using genomic sequencing technologies in refractory/recurrent tumors from children and adolescents with cancer. The NCI-COG Pediatric MATCH study (APEC1621) is considered one study under one IND consisting of a Master Version Control protocol, screening protocol component and multiple subprotocol components, with each component being contained in its own separate document. Each “component” consists of the protocol document and its associated informed consent document. The Master Version Control Protocol is the overarching administrative protocol that will keep an up-to-date record of the current version of the screening protocol component and each subprotocol component of Pediatric MATCH. Since each subprotocol component operates independently from the other subprotocol components contained in Pediatric MATCH, each has its own version date. Likewise, the screening protocol component has its own version date since changes in any of the subprotocols may not require changes in the screening protocol.

Emily Greengard
All
12 Months to 21 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03213678
STUDY00001752
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Inclusion Criteria:

• Patient must have enrolled onto APEC1621SC and must have been given a treatment assignment to Molecular Analysis for Therapy Choice (MATCH) to APEC1621D based on the presence of an actionable mutation as defined in APEC1621SC; note that treatment assignment may be to primary cohort A for patients with TSC1 or TSC2 loss of function mutations or primary cohort B for patients with other PI3K/MTOR pathway mutations
• Patients accruing to dose level 1 must have a body surface area >= 0.52 m^2 at the time of study enrollment; patients accruing to dose level 2 must have a body surface area >= 0.37 m^2 at the time of study enrollment; patients accruing to dose level -1 must have a body surface area >= 0.75 m^2 at the time of study enrollment
• Patients must have radiographically measurable disease at the time of study enrollment; patients with neuroblastoma who do not have measurable disease but have metaiodobenzylguanidine (MIBG) positive (+) evaluable disease are eligible; measurable disease in patients with CNS involvement is defined as any lesion that is at minimum 10 mm in one dimension on standard magnetic resonance imaging (MRI) or computed tomography (CT)
• Note: The following do not qualify as measurable disease:
• Malignant fluid collections (e.g., ascites, pleural effusions)
• Bone marrow infiltration except that detected by MIBG scan for neuroblastoma
• Lesions only detected by nuclear medicine studies (e.g., bone, gallium or positron emission tomography [PET] scans) except as noted for neuroblastoma
• Elevated tumor markers in plasma or cerebrospinal fluid (CSF)
• Previously radiated lesions that have not demonstrated clear progression post radiation
• Leptomeningeal lesions that do not meet the measurement requirements for Response Evaluation Criteria in Solid Tumors (RECIST) 1.1
• Bone lesions without an associated soft tissue mass >= 10 mm in greatest diameter; bone lesions with an associated soft tissue mass >= 10 mm in greatest diameter imaged by CT or MRI are considered measurable
• Karnofsky >= 50% for patients > 16 years of age and Lansky >= 50 for patients =< 16 years of age; Note: neurologic deficits in patients with CNS tumors must have been stable for at least 7 days prior to study enrollment; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
• Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment; if after the required timeframe, the numerical eligibility criteria are met, e.g. blood count criteria, the patient is considered to have recovered adequately
• Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive; >= 21 days after the last dose of cytotoxic or myelosuppressive chemotherapy (42 days if prior nitrosourea)
• Anti-cancer agents not known to be myelosuppressive (e.g. not associated with reduced platelet or absolute neutrophil count [ANC] counts): >= 7 days after the last dose of agent
• Antibodies: >= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =< 1
• Corticosteroids: if used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Hematopoietic growth factors: >= 14 days after the last dose of a long-acting growth factor (e.g. pegfilgrastim) or 7 days for short-acting growth factor; for growth factors that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur; the duration of this interval must be discussed with the study chair and the study-assigned research coordinator
• Interleukins, interferons and cytokines (other than hematopoietic growth factors): >= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors)
• Stem cell Infusions (with or without total body irradiation [TBI]):
• Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor lymphocyte infusion (DLI) or boost infusion: >= 84 days after infusion and no evidence of graft versus host disease (GVHD)
• Autologous stem cell infusion including boost infusion: >= 42 days
• Cellular therapy: >= 42 days after the completion of any type of cellular therapy (e.g. modified T cells, natural killer [NK] cells, dendritic cells, etc.)
• Radiation therapy (XRT)/external beam irradiation including protons: >= 14 days after local XRT; >= 150 days after TBI, craniospinal XRT or if radiation to >= 50% of the pelvis; >= 42 days if other substantial bone marrow (BM) radiation
• Note: radiation may not be delivered to "measurable disease" tumor site(s) being used to follow response to subprotocol treatment
• Radiopharmaceutical therapy (e.g., radiolabeled antibody, iobenguane I-131 [131I-MIBG]): >= 42 days after systemically administered radiopharmaceutical therapy
• Patients must not have received prior exposure to LY3023414
• Patients must not have received prior exposure to an agent specifically directed at the PI3K/MTOR pathway (a PI3K inhibitor, an AKT inhibitor, an MTOR inhibitor, including rapalogs, or a combined PI3K/MTOR inhibitor)
• For patients with solid tumors without known bone marrow involvement:
• Peripheral absolute neutrophil count (ANC) >= 1000/mm^3
• Platelet count >= 100,000/mm^3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment)
• Patients with known bone marrow metastatic disease will be eligible for study provided they meet the blood counts (may receive transfusions provided they are not known to be refractory to red cell or platelet transfusions); these patients will not be evaluable for hematologic toxicity
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 ml/min/1.73 m^2 or a serum creatinine based on age/gender as follows:
• Age: 1 to < 2 years; maximum serum creatinine (mg/dL): male 0.6; female 0.6
• Age: 2 to < 6 years; maximum serum creatinine (mg/dL): male 0.8; female 0.8
• Age: 6 to < 10 years; maximum serum creatinine (mg/dL): male 1; female 1
• Age: 10 to < 13 years; maximum serum creatinine (mg/dL): male 1.2; female 1.2
• Age: 13 to < 16 years; maximum serum creatinine (mg/dL): male 1.5; female 1.4
• Age: >= 16 years; maximum serum creatinine (mg/dL): male 1.7; female 1.4
• Bilirubin (sum of conjugated + unconjugated) =< 1.5 x upper limit of normal (ULN) for age
• Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135 U/L; (for the purpose of this study, the ULN for SGPT is 45 U/L)
• Serum albumin >= 2 g/dL
• Patients must have a normal blood sugar level for age; if an initial random draw (i.e. non-fasting) blood glucose value is out of range, it is acceptable to repeat this test as a fasting draw
• Patients must have a serum triglyceride level =< 300 mg/dL and serum cholesterol level =< 300 mg/dL; if an initial random draw (i.e. non-fasting) is out of range, it is acceptable to repeat this test as a fasting draw
• Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled
• Nervous system disorders (by Common Terminology Criteria for Adverse Events version
• 0 [CTCAE V 5.0]) resulting from prior therapy must be =< grade 2, with the exception of decreased tendon reflex (DTR); any grade of DTR is eligible
• Corrected QT (QTc) interval =< 480 milliseconds
• Patients must be able to swallow intact tablets
• All patients and/or their parents or legally authorized representatives must sign a written informed consent; assent, when appropriate, will be obtained according to institutional guidelines
Exclusion Criteria:

• Pregnant or breast-feeding women will not be entered on this study; pregnancy tests must be obtained in girls who are post-menarchal; males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method while receiving study treatment and for 3 months after the last dose of LY3023414
• Concomitant medications
• Corticosteroids: patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible; if used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Investigational drugs: patients who are currently receiving another investigational drug are not eligible
• Anti-cancer agents: patients who are currently receiving other anti-cancer agents are not eligible
• Anti-GVHD agents post-transplant: patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible for this trial
• Patients who have an uncontrolled infection are not eligible
• Patients who have insulin dependent diabetes are not eligible
• Patients who have received a prior solid organ transplantation are not eligible
• Patients with subependymal giant cell astrocytomas (SEGAs) are not eligible
• Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible
Other: Laboratory Biomarker Analysis, Other: Pharmacological Study, Drug: Samotolisib
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Non-Hodgkin Lymphoma, Ann Arbor Stage IV Non-Hodgkin Lymphoma, Malignant Glioma, Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Glioma, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Peripheral Primitive Neuroectodermal Tumor, Recurrent Primary Central Nervous System Neoplasm, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Germ Cell Tumor, Refractory Malignant Solid Neoplasm, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Primary Central Nervous System Neoplasm, Rhabdoid Tumor, Stage III Osteosarcoma AJCC v7, Stage III Soft Tissue Sarcoma AJCC v7, Stage IV Osteosarcoma AJCC v7, Stage IV Soft Tissue Sarcoma AJCC v7, Stage IVA Osteosarcoma AJCC v7, Stage IVB Osteosarcoma AJCC v7, Wilms Tumor
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Spinal Manipulation and Patient Self-Management for Preventing Acute to Chronic Back Pain (PACBACK) (PACBACK)

The long-term objective is to reduce overall low back pain (LBP) burden by testing scalable, first-line, non-pharmacologic strategies that address the biopsychosocial aspects of acute/sub-acute LBP and prevent transition to chronic LBP. This is a novel randomized hybrid trial addressing both effectiveness and implementation. A total of 1180 patients will be enrolled with nonspecific LBP of 2-12 weeks duration, at medium or high risk of developing chronic LBP. This multi-site, predominantly pragmatic, phase III trial has two main aims. Aim 1 will assess the effectiveness of Spinal Manipulation Therapy (SMT), Supported Self-Management (SSM), and SMT+SSM relative to Medical Care (MC) in a randomized trial using a 2x2 factorial design. Aim 2 will use mixed methods to gather data about influences on the interventions that could affect interpretation of results and future implementation.

Gert Bronfort
All
18 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT03581123
SITE00000150
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Inclusion Criteria:

• At least 18 years of age
• Acute or sub-acute low back pain
• Average low back pain severity ≥3 on the 0-10 numerical rating scale over 7 days
• Medium or high risk for persistent disabling back pain according to the STarT Back screening tool
• Ability to read and write fluently in English
Exclusion Criteria:

• Non-mechanical causes of low back pain
• Contraindications to study treatments (e.g,. surgical fusion of lumbar spine)
• Active management of current episode of low back pain by another healthcare provider
• Serious co-morbid health condition that either requires medical attention or has a risk for general health decline over the next year
• Pregnancy, current or planned during study period and nursing mothers
• Inability or unwillingness to give written informed consent
Behavioral: Supported-Self Management (SSM), Other: Spinal Manipulation Therapy (SMT), Combination Product: SMT + SSM, Drug: Standard Medical Care (SMC)
Acute Pain, Low Back Pain, Mechanical
acute/subacute low back pain, randomized clinical trial, self-management, behavioral modification, spinal manipulation therapy, standard medical care, secondary prevention
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Using Smartphone Sensor Technology to Characterize Ambulatory Patterns of Participants With Peripheral Artery Disease

Supervised exercise training (SET) improves functioning and quality of life for patients with peripheral artery disease (PAD). However, these programs have primarily been conducted in research settings, thus the physical activity that patients complete in real-world settings (urban and rural hospitals) is unknown. The proposed project will use a novel smartphone app called Daynamica, to summarize patients activity patterns and also to enhance patient-study staff communication and subsequently improve PAD patient health outcomes.

All
40 Years and over
This study is NOT accepting healthy volunteers
NCT04124315
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Inclusion Criteria:

• Diagnosis of atherosclerotic PAD and referred to hospital-based SET
• Ability to complete an evaluation of physical function and walk on a treadmill
• Resting ankle-brachial index (ABI) of ≤0.90 or stenosis ≥50% in a peripheral vessel or those with lifestyle limiting vascular-related claudication
• Those with a resting ABI of 0.91-0.99 (borderline) who have completed an exercise-ABI assessment with a >20% drop compared to resting values
• Those with ABI >1.40 who have had an abnormal toe-brachial index of ≤0.70
Exclusion Criteria:

• Lower extremity amputation(s) which interfere(s) with walking on the treadmill.
• Individuals with critical limb ischemia defined by ischemic rest pain or ischemic ulcers/gangrene on the lower extremities
• PAD of non-atherosclerotic nature (e.g., fibromuscular dysplasia, irradiation, endofibrosis)
• Females who are pregnant
• Coronary artery bypass grafts or major surgical procedures within 6 months prior to screening
• Individuals whose walking exercise is primarily limited by symptoms of chronic obstructive pulmonary disease, angina, or heart failure
• Individuals who have had a myocardial infarction within 3 months prior to screening
• Individuals who have had a transient ischemic attack or stroke 3 months prior to screening
• Individuals with uncontrolled hypertension (≥180 systolic or ≥100 diastolic resting blood pressure) during screening
• Poorly controlled diabetes defined as glycated hemoglobin >12%
• Abnormal results of blood work not conducive to safely participating in an exercise trial (e.g., anemic, electrolyte abnormalities)
• Inability to speak English
• Other clinically significant cardiovascular, pulmonary, renal, endocrine, hepatic, neurological, psychiatric, immunological, gastrointestinal, hematological, or metabolic disease that is, in the opinion of the study team, not stabilized or may otherwise confound the results of the study
Other: Accelerometry, Other: Daynamica app
Peripheral Artery Disease
peripheral artery disease, accelerometer, smartphone, supervised exercise therapy
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SLIP II Registry: Spinal Laminectomy Versus Instrumented Pedicle Screw Fusion (SLIP II)

We aim to conduct a randomized control trial comparing patient outcomes and satisfaction with or without expert panel review before making a final decision about surgery for grade I degenerative lumbar spondylolisthesis. A prospective multi-center registry aimed at addressing important issues pertaining to outcomes from treatment for degenerative spondylolisthesis and spinal stenosis will be generated.

David Polly
All
18 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT03570801
STUDY00003517
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Inclusion Criteria:

• Symptomatic lumbar spinal stenosis: defined as radicular and/or back pain either induced or aggravated by activity and relieved by rest.
• Single-level grade I degenerative spondylolisthesis (3-14mm)
Exclusion Criteria:

• Serious medical illness (ASA Class IV or higher)
• Spondylolysis
• Multilevel spondylolisthesis or high grade spondylolisthesis (grade II or greater than 14mm)
Other: Expert Panel Review
Lumbar Spondylolisthesis, Grade 1 Spondylolisthesis, Lumbar Spinal Stenosis, Degenerative Spondylolisthesis, Bone, Joint & Muscle
Lumbar Spondylolisthesis, Grade 1 Spondylolisthesis, Lumbar Spinal Stenosis, Degenerative Spondylolisthesis
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COG ALTE16C1 - Effects of Modern Chemotherapy Regimens on Spermatogenesis and Steroidogenesis in Adolescent and Young Adult (AYA) Survivors of Osteosarcoma

PRIMARY OBJECTIVES: I. Determine whether infertility and/or biomarkers of spermatogenesis and steroidogenesis differ in male osteosarcoma survivors treated with cisplatin with or without ifosfamide compared to male controls without a history of cancer. II. Evaluate whether cisplatin with or without ifosfamide for the treatment of osteosarcoma alters sperm deoxyribonucleic acid (DNA) methylation. SECONDARY OBJECTIVES: I. Evaluate the role of genetic susceptibility in the development of impairments in spermatogenesis or steroidogenesis with contemporary regimens for the treatment of osteosarcoma. OUTLINE: Participants complete a health questionnaire over 30-45 minutes. Patients also provide saliva and semen samples and undergo collection of blood.

Karim Sadak
Male
18 Years to 50 Years old
NA
This study is NOT accepting healthy volunteers
NCT03206450
STUDY00004541
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Inclusion Criteria:

• Received upfront therapies for osteosarcoma, which included cisplatin, (with or without other agents)
• Patient must have completed cancer treatment >= 2 years prior to study enrollment
• Osteosarcoma survivors without a systemically treated relapse or subsequent malignancy
• Note: History of relapse or second malignancy is permitted if treated with local therapy only (e.g. surgery, radiation)
• Able to speak, read and write in English, French or Spanish
• All patients and/or their parents or legal guardians must sign a written informed consent
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
Procedure: Biospecimen Collection, Other: Laboratory Biomarker Analysis, Other: Questionnaire Administration
Osteosarcoma
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Brivaracetam to Reduce Neuropathic Pain in Chronic SCI: A Pilot Clinical Trial

This clinical trial will determine if the drug Brivaracetam will reduce neuropathic pain in individuals with spinal cord injuries.

Leslie Morse
All
18 Years and over
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT04379011
STUDY00010959
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Inclusion Criteria:
18 years of age or older Completed inpatient rehabilitation and living in the community, Ongoing severe below-level neuropathic pain (daily average 9/10 or 10/10), Tried and failed to achieve adequate pain relief with the use of other drugs (previous pain management drugs failed to decrease their pain below a self-reported level of 9 out of 10), For people of child-bearing potential: currently practicing an effective form of two types of birth control (defined as those, alone or in combination, that result in a low failure rate (i.e., less than 1% per year) when used consistently and correctly).
Exclusion Criteria:
Progressive myelopathy secondary to posttraumatic cord tethering or syringomyelia; Active use of drugs known to interact with brivaracetam: rifampin, carbamazepine, sodium oxybate, buprenorphine, propoxyphene, levetiracetam, and phenytoin; Brain injury or cognitive impairment limiting the ability to follow directions or provide informed consent; Pregnancy or lactation; Epilepsy or active treatment for seizure disorder; Past or current suicidality; Active treatment for psychiatric disease; Drug addiction; Moderate or heavy alcohol intake (up to four alcoholic drinks for men and three for women in any single day, and a maximum of 14 drinks for men and 7 drinks for women per week); Hepatic cirrhosis, Child-Pugh grades A, B, and C; Impaired renal function; Contraindications to brivaracetam or pyrrolidine derivatives including allergy; Active clinically significant disease (e.g., renal, hepatic, neurological, cardiovascular, pulmonary, endocrine, psychiatric, hematologic, urologic, or other acute or chronic illness) that, in the opinion of the investigator, would make the patient an unsuitable candidate for this trial; History of malabsorption or other gastrointestinal (GI) disease that may significantly alter the absorption of brivaracetam; Use of any investigational drug 30 days prior to enrollment in this study.
Drug: Brivaracetam, Other: Placebo
Spinal Cord Injuries
Neuropathic Pain, Spinal Cord Injury
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A Prospective Multicenter Study Evaluating the Effect of Implant Material and/or Surface Structure on Progression of Fusion in XLIF? Surgery (AMS in XLIF)

This study is a prospective, non-concurrent, multicenter study to compare the clinical and radiographic outcomes of smooth Polyetheretherketone (PEEK), 3D-printed titanium, and Porous PEEK interbody implants when used with cancellous allograft chips with Bone Marrow Aspirate (BMA) or cellular allograft in subjects who undergo lumbar lateral interbody fusion at one or two levels. Patients will be followed up to 24 months post-surgery. Fusion rates and clinical outcomes of the 3 groups will be evaluated.

Jonathan Sembrano
All
18 Years to 80 Years old
NA
This study is NOT accepting healthy volunteers
NCT03649490
STUDY00004973
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Inclusion Criteria:

• Male and female patients who are 18-80 years of age;
• Planned interbody fusion surgery, including at least one level of extreme lateral interbody fusion (XLIF) prior to enrollment in the research at one or two consecutive lumbar levels for degenerative disc disease, including those with up to Grade 2 spondylolisthesis, with one of the following NuVasive, Inc. interbody implants:
• Coroent® XL PEEK interbody implant; or
• Modulus® 3D-printed titanium interbody implant; or
• Cohere® XLIF Porous PEEKTM interbody implant. If a transforaminal lumbar interbody fusion or anterior lumbar interbody fusion (TLIF or ALIF) is planned adjacent to the XLIF level, the same NuVasive, Inc. interbody implant material type must be used as determined by the implant enrollment schedule. For example:
• Smooth PEEK: Coroent XL PEEK (XLIF) + Coroent L (TLIF) or Coroent XLR (ALIF) or Brigade (ALIF)
• 3D-printed titanium: Modulus XLIF + Modulus TLIF or Modulus ALIF
• Porous PEEK: Cohere XLIF + Coalesce TLIF (currently no ALIF option)
• The planned procedure must include placement of bilateral posterior screw fixation with or without intrafacet fusion using autograft (with or without the assigned allograft used at the XLIF level(s)) at the treated level(s). Direct posterior decompression at the index interbody fusion level(s) is acceptable.
• Preoperative coronal Cobb angle of < 10°;
• Able to undergo surgery based on physical exam, medical history, and surgeon judgment;
• Understands the conditions of enrollment and willing to sign an informed consent to participate in the evaluation.
Exclusion Criteria:

• Use of BMP, synthetic bone graft substitutes, allografts, or any other graft material in the interbody or intrafacet spaces other than those under study;
• Posterior grafting other than the allowed intrafacet fusion at the treated level(s);
• Revision of prior fusion at treated level(s) (adjacent level interbody fusion is acceptable);
• XLIF procedure that requires or results in the release of the anterior longitudinal ligament or posterior osteotomy;
• Preoperative coronal Cobb angle of ≥ 10°;
• Procedures performed with XLIF interbody implants with integrated vertebral body screw(s);
• Active smoking six (6) weeks prior to surgery;
• Systemic or local infection (active or latent);
• Diseases that significantly inhibit bone healing (e.g., prior diagnosis of osteoporosis, metabolic bone disease, uncontrolled diabetes, dialysis dependent renal failure, symptomatic liver disease);
• Rheumatoid arthritis or other autoimmune disease that, in the option of the investigator, would interfere with bone healing and/or fusion;
• Treatment with pharmaceuticals interfering with calcium metabolism;
• Undergoing chemotherapy or radiation treatment or chronic use of steroids (defined as more than 6 weeks of steroid use within 12 months of surgery or anytime postoperatively, other than episodic use or inhaled corticosteroids);
• Use of bone stimulators postoperatively;
• Non-ambulatory, wheelchair-bound;
• Involvement in active litigation relating to the spine (worker's compensation claim is allowed if it is not contested);
• Significant general illness (e.g., HIV, active metastatic cancer of any type, uncontrolled diabetes, dialysis dependent renal failure, symptomatic liver disease);
• Spinal metastases or active spinal tumor malignancy;
• Immunocompromised or is being treated with immunosuppressive agents;
• Pregnant, or plans to become pregnant during the study;
• Mental or physical condition that would limit the ability to comply with study requirements;
• Prisoners;
• Participating in another clinical study that would confound study data.
Degenerative Disc Disease, Spondylolisthesis
Clinics and Surgery Center (CSC)
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Ivosidenib in Treating Patients With Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With IDH1 Mutations (A Pediatric MATCH Treatment Trial)

The NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) study, referred to as Pediatric MATCH, will match targeted agents with specific molecular changes identified using genomic sequencing technologies in refractory/recurrent tumors from children and adolescents with cancer. The NCI-COG Pediatric MATCH study (APEC1621) is considered one study under one IND consisting of a Master Version Control protocol, screening protocol component and multiple subprotocol components, with each component being contained in its own separate document. Each “component” consists of the protocol document and its associated informed consent document. The Master Version Control Protocol is the overarching administrative protocol that will keep an up-to-date record of the current version of the screening protocol component and each subprotocol component of Pediatric MATCH. Since each subprotocol component operates independently from the other subprotocol components contained in Pediatric MATCH, each has its own version date. Likewise, the screening protocol component has its own version date since changes in any of the subprotocols may not require changes in the screening protocol.

Emily Greengard
All
12 Months to 21 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT04195555
STUDY00001752
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Inclusion Criteria:

• Patient must have enrolled onto APEC1621SC and must have been given a treatment assignment to MATCH to APEC1621K based on the presence of an actionable mutation as defined in APEC1621SC
• Patients must be >= than 12 months and =< 21 years of age at the time of study enrollment
• Patients must have a body surface area >= 0.78 m^2 at enrollment
• Patients must be able to swallow intact tablets
• Patients must have radiographically measurable disease at the time of study enrollment. Patients with neuroblastoma who do not have measurable disease but have metaiodobenzylguanidine (MIBG)+ evaluable disease are eligible. Measurable disease in patients with CNS involvement is defined as any lesion that is at minimum 10 mm in one dimension on standard magnetic resonance imaging (MRI) or computed tomography (CT)
• Note: The following do not qualify as measurable disease:
• Malignant fluid collections (e.g., ascites, pleural effusions)
• Bone marrow infiltration except that detected by MIBG scan for neuroblastoma
• Lesions only detected by nuclear medicine studies (e.g., bone, gallium or positron emission tomography [PET] scans) except as noted for neuroblastoma
• Elevated tumor markers in plasma or cerebrospinal fluid (CSF)
• Previously radiated lesions that have not demonstrated clear progression post radiation
• Leptomeningeal lesions that do not meet the measurement requirements for Response Evaluation Criteria in Solid Tumors (RECIST) 1.1.
• Karnofsky >= 50% for patients > 16 years of age and Lansky >= 50 for patients =< 16 years of age. Note: Neurologic deficits in patients with CNS tumors must have been relatively stable for at least 7 days prior to study enrollment. Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
• Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment. If after the required timeframe, the numerical eligibility criteria are met, e.g. blood count criteria, the patient is considered to have recovered adequately
• Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive. >= 21 days after the last dose of cytotoxic or myelosuppressive chemotherapy (42 days if prior nitrosourea)
• Anti-cancer agents not known to be myelosuppressive (e.g. not associated with reduced platelet or absolute neutrophil count [ANC] counts): >= 7 days after the last dose of agent
• Antibodies: >= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =< 1
• Corticosteroids: If used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Hematopoietic growth factors: >= 14 days after the last dose of a long-acting growth factor (e.g. pegfilgrastim) or 7 days for short-acting growth factor. For growth factors that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the study chair and the study-assigned research coordinator
• Interleukins, interferons and cytokines (other than hematopoietic growth factors): >= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors)
• Stem cell infusions (with or without total body irradiation [TBI]):
• Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor lymphocyte infusion (DLI) or boost infusion: >= 84 days after infusion and no evidence of graft versus host disease (GVHD)
• Autologous stem cell infusion including boost infusion: >= 42 days
• Cellular Therapy: >= 42 days after the completion of any type of cellular therapy (e.g. modified T cells, natural killer [NK] cells, dendritic cells, etc.)
• Radiation therapy (XRT)/external beam Irradiation including protons: >= 14 days after local XRT; >= 150 days after TBI, craniospinal XRT or if radiation to >= 50% of the pelvis; >= 42 days if other substantial bone marrow (BM) radiation
• Note: Radiation may not be delivered to "measurable disease" tumor site(s) being used to follow response to subprotocol treatment
• Radiopharmaceutical therapy (e.g., radiolabeled antibody, 131I-MIBG): >= 42 days after systemically administered radiopharmaceutical therapy
• Patients must not have received prior exposure to AG-120 (ivosidenib) or other IDH1 inhibitors
• For patients with solid tumors without known bone marrow involvement: Peripheral absolute neutrophil count (ANC) >= 1000/mm^3 (within 7 days prior to enrollment)
• For patients with solid tumors without known bone marrow involvement: Platelet count >= 100,000/mm^3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment)
• Patients with known bone marrow metastatic disease will be eligible for study provided they meet the blood count (may receive transfusions provided they are not known to be refractory to red cell or platelet transfusions). These patients will not be evaluable for hematologic toxicity
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 ml/min/1.73 m^2 (within 7 days prior to enrollment), or
• A serum creatinine based on age/gender (within 7 days prior to enrollment)
• Age 1 to < 2 years, maximum serum creatinine (mg/dL) male 0.6, female 0.6
• Age 2 to < 6 years, maximum serum creatinine (mg/dL) male 0.8, female 0.8
• Age 6 to < 10 years, maximum serum creatinine (mg/dL) male 1, female 1
• Age 10 to < 13 years, maximum serum creatinine (mg/dL) male 1.2, female 1.2
• Age 13 to < 16 years, maximum serum creatinine (mg/dL) male 1.5, female 1.4
• Age >= 16 years, maximum serum creatinine (mg/dL) male 1.7, female 1.4
• Bilirubin (sum of conjugated + unconjugated) =< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment)
• Serum glutamate-pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135 U/L. (For the purpose of this study, the ULN for SGPT is 45 U/L) (within 7 days prior to enrollment)
• Serum albumin >= 2 g/dL (within 7 days prior to enrollment)
• Corrected QT (QTc )interval =< 450 milliseconds (within 7 days prior to enrollment)
• Note: Patients should avoid concomitant medication known or suspected to prolong QTc interval or cause Torsades de Pointes. Patients who are receiving drugs that prolong the QTc are eligible if the drug is necessary and no alternatives are available
• Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled
• Nervous system disorders (Common Terminology Criteria for Adverse Events [CTCAE] version [v]5.0) resulting from prior therapy must be =< grade 2, with the exception of decreased tendon reflex (DTR). Any grade of DTR is eligible
• All patients and/or their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines
Exclusion Criteria:

• Pregnant or breast-feeding women will not be entered on this study due to risks because there is yet no available information regarding human fetal or teratogenic toxicities. Pregnancy tests must be obtained in girls who are post-menarchal. Males or females of reproductive potential may not participate unless they have agreed to use an effective non-hormonal contraceptive method for the duration of study treatment and for at least 1 month after last dose of AG-120 (ivosidenib). Since AG-120 (ivosidenib) may decrease concentrations of hormonal contraceptives, hormonal contraceptives are not considered effective contraception when co-administered with AG-120 (ivosidenib)
• Patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible. If used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Patients who are currently receiving another investigational drug are not eligible
• Patients who are currently receiving other anti-cancer agents are not eligible
• Patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible for this trial
• Patients who are currently receiving drugs that are strong inducers or moderate to strong inhibitors of CYP3A4 are not eligible. Strong inducers or moderate to strong inhibitors of CYP3A4 should be avoided from 14 days prior to enrollment to the end of the study. Note: CYP3A4 inducing anti-epileptic drugs and dexamethasone for CNS tumors or metastases, on a stable dose, are allowed. In addition, patients receiving sensitive or narrow therapeutic range substrates of CYP3A4 are not eligible
• Patients with a history of progressive multifocal leukoencephalopathy are not eligible
• Patients who have an uncontrolled infection are not eligible
• Patients who have received a prior solid organ transplantation are not eligible
• Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible
Drug: Ivosidenib
Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Glioma, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Peripheral Primitive Neuroectodermal Tumor, Recurrent Rhabdoid Tumor, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Recurrent WHO Grade 2 Glioma, Refractory Ependymoma, Refractory Ewing Sarcoma, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Germ Cell Tumor, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Peripheral Primitive Neuroectodermal Tumor, Refractory Rhabdoid Tumor, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Refractory WHO Grade 2 Glioma, Wilms Tumor
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SILVIA: SIJ Stabilization in Multilevel Spine Fusion with Fixation to the Pelvis: Randomized Controlled Trial (SILVIA)

Standard multilevel fusion surgery with fixation to pelvis using S2AI screws Same + use of iFuse-3D in the “bedrock” trajectory

Christopher Martin
All
18 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT04062630
STUDY00007463
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Inclusion Criteria:

• Age 21-75 at time of screening
• Patient scheduled for multilevel (>3 levels) spinal fusion surgery with planned fixation to the pelvis using S2AI screws
• Patient has signed study-specific informed consent form
• Patient has the necessary mental capacity to participate and is physically able to comply with study protocol requirements
Exclusion Criteria:

• Indication for multilevel spine fusion surgery is any of the following:
• Congenital neuromuscular disease
• Prior pelvic fixation (i.e., patient already has S2AI or iliac bolts in place, current surgery indicated to revise this hardware)
• Grade IV spondylolisthesis
• Prior sacroiliac joint fusion/fixation on either side
• Presence of spinal cord stimulator
• Presence of severe hip pain that could impair functional and quality of life improvement from complex spine surgery
• Surgeon plans to use iliac screw for pelvic fixation
• Any known sacral or iliac pathology
• Any condition or anatomy that makes treatment with the iFuse Implant System infeasible
• Known metabolic bone disease
• Severe osteoporosis
• Known allergy to titanium or titanium alloys
• Use of medications known to have detrimental effects on bone quality and soft-tissue healing
• Neurologic condition that would interfere with postoperative physical therapy
• Current local or systemic infection that raises the risk of surgery
• Patient currently receiving or seeking short- or long-term worker's compensation and/or currently involved in injury litigation related to the SI joint or low back pain.
• Currently pregnant or planning pregnancy in the next 2 years
• Prisoner or a ward of the state.
• Known or suspected drug or alcohol abuse
• Uncontrolled psychiatric disease that could interfere with study participation
• Fibromyalgia
Device: iFuse 3-D in Bedrock Configuration, Procedure: Multilevel Lumbar Fusion surgery
Sacroiliac Joint Disruption, Scoliosis Lumbar Region, Bone, Joint & Muscle
Adult Scoliotic Spinal Deformity, Sacroiliac joint pain, multilevel lumbar surgery, SI Joint pain, Clinics and Surgery Center (CSC)
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Stability 2: ACL Reconstruction +/- Lateral Tenodesis with Patellar vs Quad Tendon (Protocol # PRO19020231) (STABILITY 2)

STABILITY 2 is a 21-site multicenter, international, randomized clinical trial that will randomly assign 1236 individuals with an anterior cruciate ligament (ACL) deficient knee who are at high risk of re-injury to anatomic anterior cruciate ligament reconstruction (ACLR) using bone patellar tendon bone (BPTB) or quadriceps tendon (QT) autograft with or without a lateral extra-articular tenodesis (LET).

Jeffrey Macalena
All
14 Years to 25 Years old
N/A
This study is NOT accepting healthy volunteers
NCT03935750
STUDY00010820
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Inclusion Criteria:

• Age 14-25,
• An ACL-deficient knee,
• Skeletal maturity (i.e. closed epiphyseal growth plates on standard knee radiographs),
• At least two of the following: participate in a competitive pivoting sport; have a pivot shift of grade 2 or greater; have generalized ligamentous laxity (Beighton score of ≥4) and/or genu recurvatum >10 degrees.
Exclusion Criteria:

• Previous ACLR on either knee,
• Partial ACL injury (defined as one bundle ACL tear requiring reconstruction/augmentation of the torn bundle with no surgery required for the intact bundle),
• Multiple ligament injury (two or more ligaments requiring surgery),
• Symptomatic articular cartilage defect requiring treatment other than debridement,
• >3 degrees of asymmetric varus,
• Inflammatory arthropathy,
• Inability to provide consent,
• Pregnancy at baseline.
Procedure: Anterior cruciate ligament reconstruction (ACLR), Procedure: Lateral extra-articular tenodesis (LET)
Anterior Cruciate Ligament Injury, Anterior Cruciate Ligament Reconstruction, Joint Instability, Bone, Joint & Muscle
Lateral extra-articular tenodesis, Autografting, Allografting, Bone-Patellar Tendon-Bone Grafting, Clinics and Surgery Center (CSC)
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Tipifarnib for the Treatment of Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With HRAS Gene Alterations, a Pediatric MATCH Treatment Trial

The NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) study, referred to as Pediatric MATCH, will match targeted agents with specific molecular changes identified using genomic sequencing technologies in refractory/recurrent tumors from children and adolescents with cancer. The NCI-COG Pediatric MATCH study (APEC1621) is considered one study under one IND consisting of a Master Version Control protocol, screening protocol component and multiple subprotocol components, with each component being contained in its own separate document. Each “component” consists of the protocol document and its associated informed consent document. The Master Version Control Protocol is the overarching administrative protocol that will keep an up-to-date record of the current version of the screening protocol component and each subprotocol component of Pediatric MATCH. Since each subprotocol component operates independently from the other subprotocol components contained in Pediatric MATCH, each has its own version date. Likewise, the screening protocol component has its own version date since changes in any of the subprotocols may not require changes in the screening protocol.

Emily Greengard
All
12 Months to 21 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT04284774
STUDY00001752
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Inclusion Criteria:

• Patient must have enrolled onto APEC1621SC and must have been given a treatment assignment to MATCH to APEC1621M based on the presence of an actionable mutation as defined in APEC1621SC
• Patients must have a body surface area >= 0.29 m^2 at enrollment
• Patients must have radiographically measurable disease at the time of study enrollment. Patients with neuroblastoma who do not have measurable disease but have metaiodobenzylguanidine (MIBG) positive (+) evaluable disease are eligible. Measurable disease in patients with CNS involvement is defined as any lesion that is at minimum 10 mm in one dimension on standard magnetic resonance imaging (MRI) or computed tomography (CT)
• Note: The following do not qualify as measurable disease:
• Malignant fluid collections (e.g., ascites, pleural effusions)
• Bone marrow infiltration except that detected by MIBG scan for neuroblastoma
• Lesions only detected by nuclear medicine studies (e.g., bone, gallium or positron emission tomography [PET] scans) except as noted for neuroblastoma
• Elevated tumor markers in plasma or cerebral spinal fluid (CSF)
• Previously radiated lesions that have not demonstrated clear progression post radiation
• Leptomeningeal lesions that do not meet the measurement requirements for Response Evaluation Criteria in Solid Tumors (RECIST) 1.1
• Karnofsky >= 50 for patients > 16 years of age and Lansky >= 50 for patients =< 16 years of age. Note: Neurologic deficits in patients with CNS tumors must have been relatively stable for at least 7 days prior to study enrollment. Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
• Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment. If after the required timeframe, the numerical eligibility criteria are met, e.g. blood count criteria, the patient is considered to have recovered adequately
• Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive.
• >= 21 days after the last dose of cytotoxic or myelosuppressive chemotherapy (42 days if prior nitrosourea)
• Anti-cancer agents not known to be myelosuppressive (e.g. not associated with reduced platelet or absolute neutrophil count [ANC] counts): >= 7 days after the last dose of agent.
• Antibodies: >= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =< 1
• Corticosteroids: If used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Hematopoietic growth factors: >= 14 days after the last dose of a long-acting growth factor (e.g. pegfilgrastim) or 7 days for short-acting growth factor. For growth factors that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the study chair and the study-assigned research coordinator
• Interleukins, interferons and cytokines (other than hematopoietic growth factors): >= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors)
• Stem cell infusions (with or without total body irradiation [TBI]):
• Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor lymphocyte infusion (DLI) or boost infusion: >= 84 days after infusion and no evidence of graft versus host disease (GVHD)
• Autologous stem cell infusion including boost infusion: >= 42 days
• Cellular therapy: >= 42 days after the completion of any type of cellular therapy (e.g. modified T cells, natural killer [NK] cells, dendritic cells, etc.)
• Radiation therapy (XRT)/external beam irradiation including protons: >= 14 days after local XRT; >= 150 days after TBI, craniospinal XRT or if radiation to >= 50% of the pelvis; >= 42 days if other substantial bone marrow (BM) radiation
• Note: Radiation may not be delivered to "measurable disease" tumor site(s) being used to follow response to subprotocol treatment
• Radiopharmaceutical therapy (e.g., radiolabeled antibody, 131I-MIBG): >= 42 days after systemically administered radio-pharmaceutical therapy
• Patients must not have received prior exposure to tipifarnib
• For patients with solid tumors without known bone marrow involvement (within 7 days prior to enrollment):
• Peripheral absolute neutrophil count (ANC) >= 1000/mm^3
• For patients with solid tumors without known bone marrow involvement (within 7 days prior to enrollment):
• Platelet count >= 100,000/mm^3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment)
• Patients with known bone marrow metastatic disease will be eligible for study provided they meet the blood counts (may receive transfusions provided they are not known to be refractory to red cell or platelet transfusions). These patients will not be evaluable for hematologic toxicity
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 ml/min/1.73 m^2 (within 7 days prior to enrollment) or
• A serum creatinine based on age/gender as follows (within 7 days prior to enrollment):
• Age: Maximum serum creatinine (mg/dL)
• 1 to < 2 years: male (0.6), female (0.6)
• 2 to < 6 years: male (0.8), female (0.8)
• 6 to < 10 years: male (1), female (1)
• 10 to < 13 years: male (1.2), female (1.2)
• 13 to < 16 years: male (1.5), female (1.4)
• >= 16 years: male (1.7), female (1.4)
• Bilirubin (sum of conjugated + unconjugated) =< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment)
• Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135 U/L. (For the purpose of this study, the ULN for SGPT is 45 U/L.) (within 7 days prior to enrollment)
• Serum albumin >= 2 g/dL (within 7 days prior to enrollment)
• Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled
• Nervous system disorders (Common Terminology Criteria for Adverse Events [CTCAE] version [v] 5.0) resulting from prior therapy must be =< grade 2
• Patients must be able to swallow intact tablets or crushed tablets mixed in water, orange juice, apple juice, tomato juice, ginger ale, applesauce, yogurt, protein shake, or a dietary supplement drink (such as Ensure). Percutaneous endoscopic gastrostomy (PEG)-tube or nasogastric tube administration is permitted
• All patients and/or their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines
Exclusion Criteria:

• Pregnant or breast-feeding women will not be entered on this study due to risks of fetal and teratogenic adverse events as seen in animal/human studies. Pregnancy tests must be obtained in girls who are post-menarchal. Males or females of reproductive potential may not participate unless they have agreed to use two effective contraceptive methods for the duration of study treatment. Both female subjects and male subjects with female partners of child-bearing potential must agree to use a highly effective method of contraception for 2 weeks prior to protocol therapy, during, and at least 4 weeks after last dose of tipifarnib. In addition, since tipifarnib could induce toxicity of male reproductive organs and cause impairment of fertility, sperm cryopreservation should be recommended for male subjects wishing to preserve their fertility following tipifarnib treatment
• Patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible. If used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Patients who are currently receiving another investigational drug are not eligible
• Patients who are currently receiving other anti-cancer agents are not eligible
• Patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible for this trial
• Patients who are currently receiving drugs that are strong inducers or inhibitors of CYP3A4/5 or UGT are not eligible. Strong inducers or inhibitors of CYP3A4/5 or UGT should be avoided from 14 days prior to the 1st dose of tipifarnib to the end of the study. In addition, patients receiving agents that are sensitive or narrow therapeutic range substrates of CYP3A4/5 are not eligible. Note: CYP3A4/5 inducing anti-epileptic drugs and dexamethasone for CNS tumors or metastases, on a stable dose, are allowed
• Patients with known hypersensitivity to tipifarnib or any components of the tablet are not eligible
• Patients with hypersensitivity to imidazoles, such as clotrimazole, ketoconazole, miconazole and others in this drug class are not eligible
• Patients who have an uncontrolled infection are not eligible
• Patients who have received a prior solid organ transplantation are not eligible
• Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible
Drug: Tipifarnib
Malignant Solid Neoplasm, Recurrent Adrenal Gland Pheochromocytoma, Recurrent Ectomesenchymoma, Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Kidney Wilms Tumor, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Glioma, Recurrent Medulloblastoma, Recurrent Melanoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Peripheral Primitive Neuroectodermal Tumor, Recurrent Rhabdoid Tumor, Recurrent Rhabdoid Tumor of the Kidney, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Recurrent Thyroid Gland Carcinoma, Recurrent WHO Grade 2 Glioma, Refractory Adrenal Gland Pheochromocytoma, Refractory Ependymoma, Refractory Ewing Sarcoma, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Germ Cell Tumor, Refractory Malignant Glioma, Refractory Medulloblastoma, Refractory Melanoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Peripheral Primitive Neuroectodermal Tumor, Refractory Rhabdoid Tumor, Refractory Rhabdoid Tumor of the Kidney, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Refractory Thyroid Gland Carcinoma, Refractory WHO Grade 2 Glioma
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COG APEC1621N - NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of LOXO-292 in Patients with Tumors Harboring RET Gene Alterations

The NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) study, referred to as Pediatric MATCH, will match targeted agents with specific molecular changes identified using genomic sequencing technologies in refractory/recurrent tumors from children and adolescents with cancer. The NCI-COG Pediatric MATCH study (APEC1621) is considered one study under one IND consisting of a Master Version Control protocol, screening protocol component and multiple subprotocol components, with each component being contained in its own separate document. Each “component” consists of the protocol document and its associated informed consent document. The Master Version Control Protocol is the overarching administrative protocol that will keep an up-to-date record of the current version of the screening protocol component and each subprotocol component of Pediatric MATCH. Since each subprotocol component operates independently from the other subprotocol components contained in Pediatric MATCH, each has its own version date. Likewise, the screening protocol component has its own version date since changes in any of the subprotocols may not require changes in the screening protocol.

Emily Greengard
All
12 Months to 21 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT04320888
STUDY00001752
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Inclusion Criteria:

• Patient must have enrolled onto APEC1621SC and must have been given a treatment assignment to MATCH to APEC1621N based on the presence of an actionable mutation
• Patients must have radiographically measurable disease at the time of study enrollment. Patients with neuroblastoma who do not have measurable disease but have metaiodobenzylguanidine (MIBG) positive (+) evaluable disease are eligible. Measurable disease in patients with CNS involvement is defined as any lesion that is at minimum 10 mm in one dimension on standard resonance imaging (MRI) or computed tomography (CT)
• Note: The following do not qualify as measurable disease:
• Malignant fluid collections (e.g., ascites, pleural effusions)
• Bone marrow infiltration except that detected by MIBG scan for neuroblastoma
• Lesions only detected by nuclear medicine studies (e.g., bone, gallium or positron emission tomography [PET] scans) except as noted for neuroblastoma
• Elevated tumor markers in plasma or cerebral spinal fluid (CSF)
• Previously radiated lesions that have not demonstrated clear progression post radiation
• Leptomeningeal lesions that do not meet the measurement requirements for Response Evaluation Criteria in Solid Tumors (RECIST) 1.1
• Karnofsky >= 50% for patients > 16 years of age and Lansky >= 50 for patients =< 16 years of age. Note: Neurologic deficits in patients with CNS tumors must have been relatively stable for at least 7 days prior to study enrollment. Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
• Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment. If after the required timeframe, the numerical eligibility criteria are met, e.g. blood count criteria, the patient is considered to have recovered adequately
• Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive.
• >= 21 days after the last dose of cytotoxic or myelosuppressive chemotherapy (42 days if prior nitrosourea)
• Anti-cancer agents not known to be myelosuppressive (e.g. not associated with reduced platelet or absolute neutrophil [ANC] counts): >= 7 days after the last dose of agent.
• Antibodies: >= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =< 1
• Corticosteroids: If used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Hematopoietic growth factors: >= 14 days after the last dose of a long-acting growth factor (e.g. pegfilgrastim) or 7 days for short-acting growth factor. For growth factors that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the study chair and the study-assigned research coordinator
• Interleukins, interferons and cytokines (other than hematopoietic growth factors): >= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors)
• Stem cell infusions (with or without total body irradiation [TBI]):
• Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor lymphocyte infusion (DLI) or boost infusion: >= 84 days after infusion and no evidence of graft versus host disease (GVHD)
• Autologous stem cell infusion including boost infusion: >= 42 days
• Cellular therapy: >= 42 days after the completion of any type of cellular therapy (e.g. modified T cells, natural killer [NK] cells, dendritic cells, etc.)
• Radiation therapy (XRT)/external beam irradiation including protons: >= 14 days after local XRT; >= 150 days after TBI, craniospinal XRT or if radiation to >= 50% of the pelvis; >= 42 days if other substantial bone marrow (BM) radiation
• Note: Radiation may not be delivered to "measurable disease" tumor site(s) being used to follow response to subprotocol treatment
• Radiopharmaceutical therapy (e.g., radiolabeled antibody, 131I-MIBG): >= 42 days after systemically administered radiopharmaceutical therapy
• Patients must not have received prior exposure to LOXO-292 or other specific RET inhibitors
• For patients with solid tumors without known bone marrow involvement (within 7 days prior to enrollment):
• Peripheral absolute neutrophil count (ANC) >= 1000/mm^3
• For patients with solid tumors without known bone marrow involvement (within 7 days prior to enrollment):
• Platelet count >= 100,000/mm^3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment)
• Patients with known bone marrow metastatic disease will be eligible for study provided they meet the blood counts (may receive transfusions provided they are not known to be refractory to red cell or platelet transfusions). These patients will not be evaluable for hematologic toxicity
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 ml/min/1.73 m^2 (within 7 days prior to enrollment) or a serum creatinine based on age/gender as follows (within 7 days prior to enrollment):
• Age: Maximum serum creatinine (mg/dL)
• 1 to < 2 years: male (0.6), female (0.6)
• 2 to < 6 years: male (0.8), female (0.8)
• 6 to < 10 years: male (1), female (1)
• 10 to < 13 years: male (1.2), female (1.2)
• 13 to < 16 years: male (1.5), female (1.4)
• >= 16 years: male (1.7), female (1.4)
• Bilirubin (sum of conjugated + unconjugated) =< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment)
• Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135 U/L. (within 7 days prior to enrollment) (For the purpose of this study, the ULN for SGPT is 45 U/L.)
• Serum albumin >= 2 g/dL (within 7 days prior to enrollment)
• Corrected QT (QTc) interval =< 480 milliseconds (within 7 days prior to enrollment)
• All patients and/or their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines
Exclusion Criteria:

• Pregnant or breast-feeding women will not be entered on this study due to risks of fetal and teratogenic adverse events as seen in animal/human studies. Pregnancy tests must be obtained in girls who are post-menarchal. Males or females of reproductive potential may not participate unless they have agreed to use two (2) highly effective contraceptive method for the duration of study treatment and for at least 2 weeks after the last dose of selpercatinib (LOXO-292). Male study participants are to refrain from sperm donation during treatment and for 2 weeks after the last dose of selpercatinib (LOXO-292)
• Patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible. If used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Patients who are currently receiving another investigational drug are not eligible
• Patients who are currently receiving other anti-cancer agents are not eligible
• Patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible for this trial
• Patients who are currently receiving drugs that are moderate or strong inducers or inhibitors of CYP3A4 are not eligible. Strong inducers or inhibitors of CYP3A4 should be avoided from 14 days prior to enrollment to the end of the study. Note: CYP3A4 inducing anti-epileptic drugs and dexamethasone for CNS tumors or metastases, on a stable dose, are allowed
• Proton pump inhibitors (PPIs), H2 receptor antagonists and antacids: Concomitant use of PPIs during selpercatinib (LOXO-292) therapy should be avoided if feasible. If co-administration of selpercatinib and PPI is necessary, administer selpercatinib with a meal. If H2 receptor antagonist is necessary, administer selpercatinib 2 hours before or 10 hours after H2 receptor antagonist administration. If antacid use is necessary, administer selpercatinib 2 or more hours before or 2 or more hours after antacid administration
• Patients who have major surgery within 14 days prior to cycle 1 day 1 (C1D1) are not eligible. (Central line placement or subcutaneous port placement is not considered major surgery)
• Patients with known clinically significant active malabsorption syndrome or other condition likely to affect gastrointestinal absorption of selpercatinib (LOXO-292) are excluded
• Patients with known hypersensitivity to any of the components of the investigational agent, LOXO 292 are excluded
• Patients with uncontrolled hypertension are excluded
• Patients with uncontrolled symptomatic hyperthyroidism and hypothyroidism (i.e. the patient required a modification to current thyroid medication in 7 days prior to enrollment) are excluded
• Patients with uncontrolled symptomatic hypercalcemia and hypocalcemia are excluded
• Patients who have an uncontrolled infection are not eligible
• Patients who have received a prior solid organ transplantation are not eligible
• Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible
Drug: Selpercatinib
Hematopoietic and Lymphoid Cell Neoplasm, Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Histiocytic and Dendritic Cell Neoplasm, Recurrent Langerhans Cell Histiocytosis, Recurrent Lymphoma, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Glioma, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Peripheral Primitive Neuroectodermal Tumor, Recurrent Rhabdoid Tumor, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Recurrent WHO Grade 2 Glioma, Refractory Ependymoma, Refractory Ewing Sarcoma, Refractory Hepatoblastoma, Refractory Histiocytic and Dendritic Cell Neoplasm, Refractory Langerhans Cell Histiocytosis, Refractory Lymphoma, Refractory Malignant Germ Cell Tumor, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Peripheral Primitive Neuroectodermal Tumor, Refractory Rhabdoid Tumor, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Refractory WHO Grade 2 Glioma, Wilms Tumor
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Identifying body awareness-related brain network changes during cognitive multisensory rehabilitation for reduced neuropathic pain in people with spinal cord injury

This mechanistic Phase II clinical trial will utilize a design with a standard parallel-arm RCT. Participants will be randomized into two groups. The Immediate Therapy Group will receive 6 weeks of CMR, 1-on-1, in-person, 3x/week, 45 min/sessions immediately, followed by 6 weeks of standard of care (no therapy) at home as a monitoring/observation period. And the Delayed Therapy Group will first complete the monitoring/observation period with 6 weeks of standard of care (no therapy) at home, followed by 6 weeks of CMR. The healthy group will not receive therapy. The baseline data obtained in the healthy control group will be compared with the baseline data and post-CMR data in both SCI groups.

Ann Van de Winckel
All
18 Years to 70 Years old
N/A
This study is also accepting healthy volunteers
NCT04706208
STUDY00008476
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Inclusion Criteria:
SCI participants:
• SCI of ≥ 3months
• medically stable with paraplegia (ASIA grade A-C, who can self-transfer with some assistance)
• neuropathic pain (>3 on the numeric pain rating scale) Able-bodied participants:
• sex and age matched
• healthy, able-bodied
Exclusion Criteria:

• MRI contra-indications (stabilizing hardware is typically MRI safe) including seizures, cognitive impairment, or other major medical complications
Behavioral: Cognitive Multisensory Therapy, Other: Usual Care, Behavioral: Clinical Assessment, Behavioral: Magnetic Resonance Imaging (MRI), Other: OPTIONAL: blood draw
Spinal Cord Injuries, Neuropathic Pain
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A Sequenced Strategy for Improving Outcomes in People With Knee Osteoarthritis Pain (SKOAP)

There is an urgent public health need to reduce our reliance on opioids for effective long-term pain management, particularly in knee osteoarthritis (KOA). This effectiveness trial will compare recommended treatments to reduce pain and functional limitations in KOA and identify clinical and patient-level factors associated with treatment response. These results will lead to improved patient selection for treatment and inform evidence based guidelines by offering well-tested, effective, non-opioid alternatives.

All
18 Years to 90 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04504812
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Inclusion Criteria:

• Meets American College of Rheumatology Classification criteria for knee osteoarthritis
Exclusion Criteria:

• Any inability to complete study procedures, including, but not limited to low English language literacy.
• Unstable medical condition that presents as an absolute or relative contraindication for participation (e.g., unstable angina, poorly controlled diabetes mellitus, end stage renal failure, automated implantable cardioverter-defibrillator that cannot be disabled before RFA).
• Severe untreated bleeding disorder (anticoagulants may be continued during phase II treatments in most patients)
• Severe vision or hearing impairment or serious cognitive impairment that could interfere with consent or outcome assessment
• Poorly controlled serious psychiatric condition
• Active substance abuse
• Scheduled joint replacement on the affected knee
• History of unilateral total knee arthroplasty (TKA) with complaints of KOA pain limited to the operated knee
• Ulcers or an open wound in the region of the index knee
Drug: Duloxetine, Combination Product: Intra-Articular Injection, Procedure: Nerve Procedure with long acting blocks, Procedure: Nerve Procedure with nerve ablation, Behavioral: Pain Coping Skills Training, Other: Best Practices
Knee Osteoarthrosis
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A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD)

Double Blind Phase 1b Study to assess ASP0367 in pediatric male patients with Duchenne Muscular Dystrophy

Male
8 Years to 16 Years old
Phase 1
This study is NOT accepting healthy volunteers
NCT04184882
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Inclusion Criteria:

• Subject has a diagnosis of Duchenne muscular dystrophy (DMD) (confirmed by Central Genetic Counselor) defined as a clinical picture consistent with typical DMD and 1 of the following:
• Dystrophin immunofluorescence and/or Western blot showing severe dystrophin deficiency consistent with the diagnosis of DMD.
• Identifiable mutation within the DMD gene (deletion/duplication of 1 or more exons), where reading frame can be predicted as "out-of-frame"
• Complete dystrophin gene sequencing showing an alteration (point mutation, duplication or other) that is expected to preclude production of the functional dystrophin protein (i.e., nonsense mutation or deletion/duplication leading to a downstream stop codon).
• A male subject of reproductive potential (Tanner Stage 2 and above) must agree to do either of the following from screening throughout the study until 30 days after the last dose of the investigational product (IP):
• Abstain from sexual intercourse, OR
• If having heterosexual intercourse, must use a condom and their female partners who are of childbearing potential must use a highly effective contraception method.
• Subject has been on a stable regimen of corticosteroids for 6 months prior to the time of enrollment (at baseline).
• Subject has been on stable cardiac therapy for 3 months prior to the time of enrollment (at baseline), if used, which may include prophylactic angiotensin-converting enzyme inhibitors (ACE), angiotensin II receptor blocker (ARB), aldosterone receptor antagonists (e.g., spironolactone, eplerenone), and/or beta-blocker therapy or a combination therapy thereof.
• Subject is unable to complete the 10 meter run/walk in <6 seconds at screening.
• Subject has a PUL 2.0 entry item A score of 4, 5 or 6 at screening.
• Subject and subject's parent(s) or legal guardian agrees not to participate in another interventional study while participating in the present study.
• For those subjects receiving exon-skipping therapy, the subject has been on a stable dose regimen with a single commercially-available product for at least 6 months prior to randomization at baseline.
• For those subjects using metformin, the subject has been on a stable dose of metformin for 3 months prior to the time of enrollment (at baseline) and the investigator expects the subject to maintain the current metformin dose.
Exclusion Criteria:

• Subject has had an acute illness (i.e., upper respiratory or viral infection) within 4 weeks prior to study enrollment (at baseline), which precludes participation.
• Subject has a cardiac ejection fraction < 55% on echocardiogram at screening.
• Subject has a mean QT interval from triplicate electrocardiogram (ECG) using Fridericia's correction (QTcF) of > 450 msec at screening. If the mean QTcF exceeds the limits stated above, 1 additional triplicate ECG can be taken and utilized at screening.
• Subject has cardiac troponin I (cTnI) above the upper limit of normal (ULN) at screening and is assessed clinically significant.
• Subject has used coenzyme Q10 (CoQ10), idebenone, carnitine, or other mitochondrial focused supplements or drugs within 4 weeks prior to randomization at baseline. In addition, subject has used any peroxisome proliferator-activated receptors (PPAR) ligands such as fibrates and thiazolidinediones 4 weeks prior to randomization at baseline.
• Subject has a known or suspected hypersensitivity to ASP0367, or any components of the formulation used.
• Subject has inadequate renal function, as defined by serum Cystatin C > 2 x ULN at screening.
• Subject who has any of the following liver function tests elevated: gamma-glutamyl transferase [GGT] and/or total bilirubin [TBL]) > 1.5 x ULN at screening.
• Subject who has a positive test result for hepatitis A virus (HAV) antibodies (immunoglobulin M [IgM]), hepatitis B surface (HBs) antigen, hepatitis C virus (HCV) antibody or human immunodeficiency virus (HIV) antigen/antibody at screening.
• Subject has mental conditions such as schizophrenia, bipolar disorder or major depressive disorder.
• Subject has a history of suicide attempt, suicidal behavior or has any suicidal ideation within 1 year prior to screening that meets criteria at a level of 4 or 5 by using the Columbia Suicide Severity Rating Scale (C-SSRS) or who is at significant risk to commit suicide, as assessed at screening or at baseline.
• Subject has severe behavioral or cognitive problems that preclude participation in the study.
• Subject has any condition, which makes the subject unsuitable for study participation.
• Subject is taking any other investigational therapy currently or has taken any other investigational therapy within 3 months prior to the time of enrollment (at baseline).
• Subject and parent/guardian are unwilling and unable to comply with scheduled visits, IP administration plan and study procedures.
• Subject has had clinical signs and symptoms consistent with coronavirus (SARSCoV-2) infection or who has tested positive within 2 months prior to randomization at baseline.
• Subject whose parent(s) and/or caregiver(s) have increased risk of coronavirus (SARS-CoV-2) exposure from work history (e.g., nursing home, meat processing facility and correctional facility) or recent travel history unless the subject's parent(s) and/or caregiver(s) have been appropriately vaccinated with one of the COVID-19 vaccines.
Drug: Bocidelpar, Drug: Placebo
Duchenne Muscular Dystrophy (DMD)
tolerability, ASP0367, MA-0211, safety
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Brain Mechanisms of Qigong for Neuropathic Pain Relief in Adults With Spinal Cord Injury

The researchers aim to prove that Qigong practice can result in reduced or relieved neuropathic pain, improved mood, life satisfaction, self-efficacy, enjoyment to move, and community integration; and decreased fear of movement, use of medication or health care services for adults with spinal cord injury.

All
18 Years to 75 Years old
N/A
This study is NOT accepting healthy volunteers
NCT04917107
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Inclusion Criteria:

• Spinal cord injury of greater than or equal to 3 months
• Medically stable with paraplegia (T1 and below) or tetraplegia (C4 and below)
• Highest level of below-level SCI-related neuropathic pain >3 on the numeric pain rating scale.
Exclusion Criteria:

• MRI contra-indications (stabilizing hardware is typically MRI safe)
• Uncontrolled seizure disorder; cognitive impairment and/or communicative disability (e.g., due to brain injury) that prevent the participant from following directions or from learning
• Ventilator dependency
• Pregnancy to plans to become pregnant during study
• Inability to perform kinesthetic imagery.
• Participants who cannot feel index finger movements will not perform the robot task but will perform all other resting-state and tasks in the MRI scanner.
Behavioral: Qigong
Spinal Cord Injuries
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Hmong Microbiome ANd Gout, Obesity, Vitamin C (HMANGO-C)

All
18 Years and over
N/A
This study is also accepting healthy volunteers
NCT04938024
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Inclusion Criteria:

• Self-identified Hmong persons whose both parents are Hmong, with and without hyperuricemia (serum urate (UA) ≥ 6.8 mg/dL ) and/or gout (defined by 2020 American College of Rheumatology Guideline for the Management of Gout) are eligible for the study.
• For those with gout, participants qualify if they have serum UA ≥ 6.8 mg/dL based on the baseline measurement or serum UA < 6.8 mg/dL based on the baseline measurement with at least 1 episode of peripheral joint or bursal swelling, pain, or tenderness (acute gout flare) in their lifetime, (with or without urate-lowering therapy)
Exclusion Criteria:

• Allergy or sensitivity to vitamin C
• Diagnosis/history of:
• Gastrointestinal surgery including colectomy, ileectomy, and gastrectomy
• Inflammatory bowel disease
• Auto-immune disease
• Type I diabetes mellitus
• Severe kidney disease (i.e., on dialysis)
• End-stage liver disease (i.e. cirrhosis)
• Glucose-6-phosphate dehydrogenase deficiency, (due to increased bleeding risk in those with G6PD deficiency when receiving vitamin C)
• Pregnant or breastfeeding persons
• Current use of:
• Antibiotics
• Probiotics supplement
• Ketogenic diet
Dietary Supplement: Vitamin C
Hyperuricemia, Gout
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UP0085: A POSTMARKETING, MULTICENTER, LONGITUDINAL, PROSPECTIVE, PHARMACOKINETIC, PHASE 1B STUDY IN PREGNANT WOMEN WITH CHRONIC INFLAMMATORY DISEASES TREATED WITH CIMZIA? (CERTOLIZUMAB PEGOL)

A postmarketing, multicenter, longitudinal, prospective, pharmacokinetic, phase 1B study in pregnant women with chronic inflammatory diseases treated with cimzia

Eugenia Shmidt
Phase IV
This study is NOT accepting healthy volunteers
NCT04163016
STUDY00009260
Digestive & Liver Health, Axial Spondyloarthritis, Crohn's Disease, Plaque Psoriasis, Psoriatic Arthritis, Rheumatoid Arthritis
inflammatory bowel disease, Axial Spondyloarthritis (AxSpA), CZP, Certolizumab Pegol, Cimzia, Clinics and Surgery Center (CSC), Crohn's Disease (CD), Pharmacokinetics, Plaque Psoriasis (PSO), Pregnant Women, Psoriatic Arthritis (PsA), Rheumatoid Arthritis (RA)
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PROmote weight loss in obese PAD patients to preVEnt mobility Loss: The PROVE Trial

More than 65% of people with lower extremity peripheral artery disease (PAD) are overweight or obese. Overweight or obese people with PAD have greater functional impairment and faster functional decline than normal weight people with PAD. Walking exercise is first line therapy to improve walking performance in PAD. However, our NHLBI-funded observational longitudinal study of functional decline in PAD showed that overweight and obese PAD participants who combined weight loss with walking exercise had significantly less functional decline than those who walked for exercise but did not lose weight. Therefore, we hypothesize that among people with PAD who are overweight or obese, a weight loss intervention combined with exercise will improve walking ability more than exercise alone. However, the effects of intentional weight loss in overweight or obese people with PAD are unknown and may not be beneficial if weight loss exacerbates PAD-related sarcopenia. Behavior change that achieves sustained weight loss is particularly challenging in older obese people with chronic disease. Therefore, among people with PAD and BMI>28 kg/m2, we will conduct a randomized clinical trial to test the hypothesis that a weight loss intervention combined with walking exercise achieves greater improvement in functional performance than exercise alone at 12-month follow-up.

Diane Treat-Jacobson
NA
This study is NOT accepting healthy volunteers
NCT04228978
STUDY00006194
Overweight or Obesity, Peripheral Artery Disease
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A community-based exercise program to improve walking outcomes in patients with peripheral artery disease

Patients with peripheral artery disease (PAD) have significant walking impairment and a decreased quality of life. This is in part due to the classic symptom of PAD called claudication. Patients describe claudication as pain, aching, and cramping in the leg muscles during walking that goes away with rest. Walking exercise in supervised hospital settings is a primary therapy, however, these programs are unavailable due to a number of issues (e.g., no reimburesement, proximity to clinics). Programs of exercise in community settings are ineffective, largely due to primarily advice given by providers for patients to go home and walk. Thus, successful community-based walking exercise programs for PAD are needed. We will test the hypothesis that PAD patients randomized to an exercise program in the community setting incorporating training, monitoring, and coaching (TMC) components commonly used in supervised exercise programs will improve peak walking time (PWT) (the length of time a patient can walk on a graded treadmill) compared with control group patients who only receive the standard of care (upfront advice to walk). Secondary hypotheses include a significant improvement in patient perceived quality of life and a significant increase in volume of physical activity for patients who complete community-based walking exercise when compared with controls. Exploratory hypotheses include an improvement in PWT for patients who receive community exercise in addition to revascularization (a procedure that opens up the arteries of the legs; a standard treatment composed of endovascular therapy or open surgery for PAD provided separately from this study). Specifically, we will explore 1) patients receiving a combination of endovascular therapy (e.g., stenting, angioplasty) and community-based walking exercise, 2) open surgery and community-based walking exercise and compare outcomes to 3) patients who do not receive endovascular therapy or open intervention regardless of randomization to exercise or control groups.

Ryan Mays
Clinical Outcomes Research
This study is NOT accepting healthy volunteers
NCT02075502
1612M01961
Peripheral Artery Disease
claudication, community walking exercise, community-based participatory research, endovascular therapy, exercise adherence
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Integrating Fall Prevention Balance Exercises Into a Program for Older Adults With Peripheral Artery Disease (PAD): A Mixed Methods Feasibility Study

All
60 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT05090774
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Inclusion Criteria:

• Enrolled in supervised exercise therapy (SET)
• One or more fall risk factors as determined by CDC STEADI fall risk screener
• One or more falls in the last year
• Unsteadiness when standing or walking
• Worries about falling
• Have symptomatic peripheral artery disease (PAD) with objective diagnosis (ABI less than or equal to 0.90 or previous revascularization)
Exclusion Criteria:

• Formal diagnosis of neurocognitive impairment or <3 on Callahan 6-Item Screener
• Current uncontrolled vestibular dysfunction (e.g. Meniere's Disease, benign paroxysmal positional vertigo)
• Participants who self-report the following symptoms will require clearance from a primary provider (as guided by the Exercise and Screening for You Questionnaire):
• Pain, tightness or pressure in chest during physical activity (walking, climbing stairs, household chores, similar activities) that have not been checked and/ or treated by a healthcare provider
• Current dizziness that have not been checked and/ or treated by a healthcare provider
• Current, frequent falls that have not been checked and/ or treated by a healthcare provider
• Other clinically significant disease that is, in the opinion of the study team, not stabilized or may otherwise confound the results of the study
Behavioral: Adapted Otago Exercise Program (OEP)
Fall, Fall Injury, Peripheral Artery Disease, Peripheral Arterial Disease
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PEPN2011 - A Phase 1/2 Study of Tegavivint (IND#156033, NSC#826393) in Children, Adolescents, and Young Adults with Recurrent or Refractory Solid Tumors, Including Lymphomas and Desmoid Tumors

This phase I/II trial evaluates the highest safe dose, side effects, and possible benefits of tegavivint in treating children, adolescents, and young adults with recurrent or refractory solid tumors, including lymphomas and desmoid tumors.

Emily Greengard
All
12 Months to 30 Years old
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT04851119
STUDY00014319
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Inclusion Criteria:

• PART A: Patients must be >= 12 months and =< 21 years of age at the time of study enrollment
• PART B: Patients must be >= 12 months and =< 30 years of age at the time of study enrollment
• Patients with recurrent or refractory solid tumors including non-Hodgkin lymphoma and desmoid tumors are eligible. Patients must have had histologic verification of malignancy at original diagnosis or relapse
• PART A: Patients with relapsed or refractory solid tumors, including patients with non-Hodgkin lymphoma and desmoid tumors
• PART B: Patients with recurrent or refractory Ewing sarcoma, desmoid tumors, osteosarcoma, liver tumors (HCC and hepatoblastoma), Wilms tumor, and tumors with Wnt pathway aberrations. For the Wnt pathway aberrations cohort we will include the most common CTNNB1 mutations (S37F, S45F, T41A, S45P, S33C, S37C, D32Y, S33F, T41I, G34R, G34V, D32N, S33P, G34E, D32G) as well as any loss of function mutations in the APC, Axin2FBXW7, TCF7L2, and RNF43 genes or any gain-of-function mutations in the GSK3B, LRP6, and LGR5 genes. For patients without prior sequencing, immunohistochemistry (IHC), is required. IHC showing strong nuclear beta-catenin staining will be accepted for the following tumor types: colorectal carcinoma, melanoma, endometrial cancer, ovarian cancer, neuroblastoma, non-Hodgkin lymphoma, pancreatic ductal adenocarcinoma, and solid pseudopapillary tumor of the pancreas
• PART A: Patients must have either measurable or evaluable disease. For desmoid tumors, the patient must have disease that the investigator deems unresectable or sufficiently morbid or potentially life-threatening that there is favorable risk/benefit to the patient to participate in the trial
• PART B: Patients must have measurable disease. For desmoid tumors, the patient must have measurable disease that the investigator deems unresectable or sufficiently morbid or potentially life-threatening that there is favorable risk/benefit to the patient to participate in the trial
• Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1 or 2. Use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age
• Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment. If after the required timeframe, the numerical eligibility criteria are met, e.g., blood count criteria, the patient is considered to have recovered adequately.
• Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive
• Solid tumor patients: >= 21 days after the last dose of myelosuppressive chemotherapy (42 days if prior nitrosourea)
• Non-Hodgkin lymphoma patients
• A waiting period prior to enrollment is not required for patients receiving standard maintenance chemotherapy (i.e., corticosteroid, vincristine, thioguanine [6MP], and/or methotrexate)
• >= 14 days must have elapsed after the completion of other cytotoxic therapy, with the exception of hydroxyurea, for patients not receiving standard maintenance therapy
• NOTE: Cytoreduction with hydroxyurea must be discontinued >= 24 hours prior to the start of protocol therapy
• Anti-cancer agents not known to be myelosuppressive (e.g., not associated with reduced platelet or absolute neutrophil counts [ANC]): >= 7 days after the last dose of agent
• Antibodies: >= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =< 1
• Corticosteroids: If used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Hematopoietic growth factors: >= 14 days after the last dose of a long-acting growth factor (e.g., pegfilgrastim) or 7 days for short acting growth factor. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur
• Interleukins, interferons and cytokines (other than hematopoietic growth factors): >= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors)
• Stem cell Infusions (with or without total-body irradiation [TBI]):
• Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor lymphocyte infusion (DLI) or boost infusion: >= 84 days after infusion and no evidence of graft versus host disease (GVHD)
• Autologous stem cell infusion including boost infusion: >= 42 days.
• Cellular therapy: >= 42 days after the completion of any type of cellular therapy (e.g., modified T cells, natural killer [NK] cells, dendritic cells, etc.).
• External beam radiation therapy (XRT)/external beam irradiation including protons: >= 14 days after local XRT; >= 150 days after TBI, craniospinal XRT or if radiation to >= 50% of the pelvis; >= 42 days if other substantial bone marrow (BM) radiation
• Radiopharmaceutical therapy (e.g., radiolabeled antibody, iobenguane I-131 [131I MIBG]): >= 42 days after systemically administered radiopharmaceutical therapy
• Patients must not have received prior exposure to tegavivint
• PATIENTS WITH SOLID TUMORS WITHOUT KNOWN BONE MARROW INVOLVEMENT: Peripheral absolute neutrophil count (ANC) >= 1000/uL (within 7 days prior to enrollment)
• PATIENTS WITH SOLID TUMORS WITHOUT KNOWN BONE MARROW INVOLVEMENT: Platelet count >= 100,000/uL(transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment) (within 7 days prior to enrollment)
• PATIENTS WITH SOLID TUMORS WITHOUT KNOWN BONE MARROW INVOLVEMENT: Hemoglobin >= 8.0 g/dL at baseline (may receive red blood cell [RBC] transfusions) (within 7 days prior to enrollment)
• Patients with known bone marrow metastatic disease will be eligible for study provided they meet blood counts (may receive transfusions provided they are not known to be refractory to red cell or platelet transfusions). These patients will not be evaluable for hematologic toxicity. At least 5 of every cohort of 6 patients must be evaluable for hematologic toxicity for the dose-escalation part of the study. If dose-limiting hematologic toxicity is observed, all subsequent patients enrolled on Part A must be evaluable for hematologic toxicity
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or a creatinine based on age/gender as follows:
• Age; maximum serum creatinine
• Age 1 to < 2 years; 0.6 mg/dL (male); 0.6 mg/dL (female)
• Age 2 to < 6 years; 0.8 mg/dL (male); 0.8 mg/dL (female)
• Age 6 to < 10 years; 1 mg/dL (male); 1 mg/dL (female)
• Age 10 to < 13 years; 1.2 mg/dL (male); 1.2 mg/dL (female)
• Age 13 to < 16 years; 1.5 mg/dL (male); 1.4 mg/dL (female)
• Age >= 16 years; 1.7 mg/dL (male); 1.4 mg/dL (female)
• PATIENTS WITH SOLID TUMORS: Bilirubin (sum of conjugated + unconjugated or total) =<
• 5 x upper limit of normal (ULN) for age
• PATIENTS WITH SOLID TUMORS: Serum glutamic pyruvic transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135 U/L. For the purpose of this study, the ULN for SGPT is 45 U/L
• PATIENTS WITH SOLID TUMORS: Albumin >= 2 g/dL
Exclusion Criteria:

• Pregnant or breast-feeding women will not be entered on this study because there is yet no available information regarding human fetal or teratogenic toxicities. Pregnancy tests must be obtained in girls who are post-menarchal. Males or females of reproductive potential may not participate unless they have agreed to use two effective methods of birth control, including a medically accepted barrier or contraceptive method (e.g., male or female condom) for the duration of the study. Abstinence is an acceptable method of birth control
• Patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible. If used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Patients who are currently receiving another investigational drug are not eligible
• Patients who are currently receiving other anti-cancer agents are not eligible
• Patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible for this trial
• Patients who are currently receiving drugs that are strong inducers or inhibitors of CYP3A4 are not eligible. Strong inducers or inhibitors of CYP3A4 should be avoided from 14 days prior to the 1st dose of tegavivint to the end of the study
• Patients who have received bisphosphonates within 4 weeks prior to study enrollment will be excluded
• Patients who have received denosumab within 180 days prior to study enrollment will be excluded
• Patients with primary brain tumors are ineligible
• Patients with known central nervous system (CNS) metastasis will be excluded
• Patients with a known metabolic bone disease (ex: hyperparathyroidism, Paget's disease, osteomalacia) are not eligible
• Patients with a disorder associated with abnormal bone metabolism will be excluded
• Patients with grade >= 2 hypocalcemia that is not corrected with oral calcium supplementation will be excluded
• Patients with vitamin D < 20 ng/mL will require supplementation, or will otherwise be excluded. Patients must agree to take vitamin D +/- calcium supplements (if necessary) according to institutional or published guidelines. Additional calcium supplementation is not required if adequate dietary intake can be ascertained
• Patients with pre-existing grade 3 osteoporosis are excluded
• Patients who have an uncontrolled infection are not eligible
• Patients who have received a prior solid organ transplantation are not eligible
• Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible
Drug: Tegavivint
Colorectal Carcinoma, Endometrial Carcinoma, Melanoma, Neuroblastoma, Ovarian Carcinoma, Pancreatic Ductal Adenocarcinoma, Recurrent Desmoid Fibromatosis, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Hepatocellular Carcinoma, Recurrent Malignant Solid Neoplasm, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Refractory Desmoid Fibromatosis, Refractory Ewing Sarcoma, Refractory Hepatoblastoma, Refractory Hepatocellular Carcinoma, Refractory Malignant Solid Neoplasm, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Solid Pseudopapillary Neoplasm of the Pancreas, Wilms Tumor
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