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ACNS1821: A Phase 1/2 Trial of Selinexor (KPT-330) and Radiation Therapy in Newly-Diagnosed Pediatric Diffuse Intrinsic Pontine Glioma (DIPG) and High-Grade Glioma (HGG)
Christopher Moertel, MD
All
12 Months to 21 Years old
STUDY0016411
STUDY0016411
Inclusion Criteria:
STEP 0: Patients must be >= 12 months and =< 21 years of age at the time of enrollment on Step 0.
Please note:
This age range includes pre-screening for all HGG patients. Individual treatment protocols may have different age criteria.
Non-DIPG patients with tumors that do not harbor an H3K27M-mutation and are >= 18 years of age will not be eligible to enroll on ACNS1821 (Step 1).
STEP 0: Patient is suspected of having localized, newly diagnosed HGG, excluding metastatic disease, OR patient has an institutional diagnosis of DIPG
STEP 0:
For patients with non-pontine tumors: Patient and/or their parents or legal guardians have signed informed consent for eligibility screening on APEC14B1 Part A.
For patients with DIPG: Patient and/or their parents or legal guardians have signed informed consent for ACNS1821.
STEP 0:
For patients with non-pontine tumors only, the specimens obtained at the time of diagnostic biopsy or surgery must be submitted through APEC14B1 ASAP, preferably within 5 calendar days of definitive surgery
STEP 1: Patients must be >= 12 months and =< 21 years of age at the time of enrollment
STEP 1: Patients must have newly-diagnosed DIPG or HGG (including DMG).
STEP 1: Stratum DIPG
Patients with newly-diagnosed typical DIPG, defined as tumors with a pontine epicenter and diffuse involvement of at least 2/3 of the pons on at least 1 axial T2 weighted image, are eligible. No histologic confirmation is required.
Patients with pontine tumors that do not meet radiographic criteria for typical DIPG (e.g., focal tumors or those involving less than 2/3 of the pontine cross-sectional area with or without extrapontine extension) are eligible if the tumors are biopsied and proven to be high-grade gliomas (such as anaplastic astrocytoma, glioblastoma, high-grade glioma not otherwise specified [NOS], and/or H3 K27M-mutant) by institutional diagnosis.
STEP 1: Stratum DMG (with H3 K27M mutation)
Patients must have newly-diagnosed non-pontine H3 K27M-mutant HGG without BRAF V600 or IDH1 mutations as confirmed by Rapid Central Pathology and Molecular Screening Reviews performed on APEC14B1
Note: Patients need not have either measurable or evaluable disease, i.e., DMG patients may have complete resection of their tumor prior to enrollment. Primary spinal tumors are eligible for enrollment. For rare H3 K27M-mutant HGG in non-midline structures (e.g., cerebral hemispheres), these patients will be considered part of Stratum DMG.
STEP 1: Stratum HGG (without H3 K27M mutation)
Patients must have newly-diagnosed non-pontine H3 K27M-wild type HGG without BRAF V600 or IDH1 mutations as confirmed by Rapid Central Pathology and Molecular Screening Reviews performed on APEC14B1
Please note:
Patients who fall in this category and who are >= 18 years of age are not eligible due to another standard-of-care regimen (radiation/temozolomide) that is available
Patients need not have either measurable or evaluable disease, i.e., HGG patients may have complete resection of their tumor prior to enrollment. Primary spinal tumors are eligible for enrollment
STEP 1: 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 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.
STEP 1: Peripheral absolute neutrophil count (ANC) >= 1000/uL (within 7 days prior to step 1 enrollment)
STEP 1: Platelet count >= 100,000/uL (transfusion independent) (within 7 days prior to step 1 enrollment)
STEP 1: Hemoglobin >= 8.0 g/dL (may receive red blood cell [RBC] transfusions) (within 7 days prior to step 1 enrollment)
STEP 1: Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 (within 7 days prior to step 1 enrollment) or
A serum creatinine based on age/gender as follows (within 7 days prior to step 1 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
STEP 1: Total bilirubin =< 1.5 x upper limit of normal (ULN) for age
STEP 1: 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.
STEP 1: Serum amylase =< 1.5 x ULN
STEP 1: Serum lipase =< 1.5 x ULN
STEP 1: No evidence of dyspnea at rest, no exercise intolerance, and a pulse oximetry > 94% if there is clinical indication for determination.
STEP 1: Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled.
STEP 1: Patients must be enrolled and protocol therapy must begin no later than 31 days after the date of radiographic diagnosis (in the case of non-biopsied DIPG patients only) or definitive surgery, whichever is the later date (Day 0).
For patients who have a biopsy followed by resection, the date of resection will be considered the date of definitive diagnostic surgery. If a biopsy only was performed, the biopsy date will be considered the date of definitive diagnostic surgery.
Exclusion Criteria:
STEP 1: Patients must not have received any prior therapy for their central nervous system (CNS) malignancy except for surgery and steroid medications.
STEP 1: Patients who are currently receiving another investigational drug are not eligible.
STEP 1: Patients who are currently receiving other anti-cancer agents are not eligible.
STEP 1: Patients >=18 years of age who have H3 K27M-wild type HGG.
STEP 1: Patients who have an uncontrolled infection.
STEP 1: Patients who have received a prior solid organ transplantation.
STEP 1: Patients with grade > 1 extrapyramidal movement disorder.
STEP 1: Patients with known macular degeneration, uncontrolled glaucoma, or cataracts.
STEP 1: Patients with metastatic disease are not eligible; MRI of spine with and without contrast must be performed if metastatic disease is suspected by the treating physician.
STEP 1: Patients with gliomatosis cerebri type 1 or 2 are not eligible, with the exception of H3 K27M-mutant bithalamic tumors.
STEP 1: Patients who are not able to receive protocol specified radiation therapy.
STEP 1:
Female patients who are pregnant are ineligible since there is yet no available information regarding human fetal or teratogenic toxicities.
Lactating females are not eligible unless they have agreed not to breastfeed their infants. It is not known whether selinexor is excreted in human milk.
Female patients of childbearing potential are not eligible unless a negative pregnancy test result has been obtained.
Sexually active patients of reproductive potential are not eligible unless they have agreed to use two effective methods of birth control (including a medically accepted barrier method of contraception, e.g., male or female condom) for the duration of their study participation and for 90 days after the last dose of selinexor. Abstinence is an acceptable method of birth control. Biopsy, Magnetic Resonance Imaging, Radiation Therapy, Selinexor, Biopsy, Magnetic Resonance Imaging, Radiation Therapy, Selinexor
Anaplastic Astrocytoma, Anaplastic Astrocytoma, Not Otherwise Specified, Diffuse Intrinsic Pontine Glioma, Diffuse Midline Glioma, H3 K27M-Mutant, Glioblastoma, Glioblastoma, Not Otherwise Specified, Malignant Glioma
COG ALTE2031 - StepByStep: A Randomized Trial of a Mobile Health and Social Media Physical Activity Intervention among Adolescent and Young Adult Childhood Cancer Survivors
The study objectives are to compare the effect of 2 different physical activity programs on physical activity levels in adolescents and young adults who received and completed treatment for cancer and to measure lab tests associated with heart health and information collected from surveys to learn how changes in physical activity levels affect health and quality of life in participants.
Lucie Turcotte
All
15 Years to 20 Years old
Phase 3
STUDY00013015
STUDY00013015
Inclusion Criteria:
First diagnosis of malignant neoplasm (International Classification of Diseases for Oncology [ICD-O] behavior code of "3") in first and continuous remission at the time of enrollment
Curative cancer treatment must have included chemotherapy (including cellular therapy) and/or radiation (including radioactive iodine)
Note: Childrens Oncology Group (COG) therapeutic trial participation is not required
All cancer treatment must have been completed within 3-36 calendar months prior to enrollment
Patients must have a life expectancy of > 1 year
Self-report of < 420 minutes of moderate-to-vigorous physical activity per week as assessed via the study-specific Physical Activity Worksheet
Note: See COG Study Web Page for the Godin-Shephard Leisure Time Physical Activity Questionnaire or link to online calculator
Ambulatory and no known medical contraindications to increasing physical activity
Note: Patients with amputation, rotationplasty, or other prothesis are not automatically excluded as long as they are ambulatory and have no known medical contraindications to increasing physical activity and all other eligibility criteria are satisfied
No known significant physical or cognitive impairment that would prevent use of the electronic devices used for the protocol intervention (e.g. Fitbit, smartphone, tablet, or computer)
Able to read and write English
Note: For patients < 18 years, consenting parent/legal guardian does not have to be able to read and write English
All patients and/or their parents or legal guardians must sign a written informed consent
Note: Informed consent may be obtained electronically/online if allowed by local site policy and Institutional Review Board (IRB)/Research Ethics Board (REB) of record
All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
Exclusion Criteria:
Post-menarchal female patients who are pregnant or planning to become pregnant in the next year are excluded
Note: Pregnancy status can be established by clinical history with patient. Post-menarchal female patients are eligible as long as they agree to use an effective contraceptive method (including abstinence) during study participation
Patients with previous hematopoietic stem cell transplant (HSCT) are excluded
Note: Patients with previous autologous HSCT, chimeric antigen receptor T-cell (CAR T-cell) therapy, and other cellular cancer therapies can participate as long as all other eligibility criteria are satisfied Educational Intervention, FitBit, Goal Setting, Health Promotion and Education, Media Intervention, Telephone-Based Intervention, Educational Intervention, FitBit, Goal Setting, Health Promotion and Education, Media Intervention, Telephone-Based Intervention
Hematopoietic and Lymphoid Cell Neoplasm, Malignant Solid Neoplasm
Defining Clinical Endpoints in LGMD (GRASP-01-001)
Peter Kang
All
4 Years to 65 Years old
SITE00001632
SITE00001632
Inclusion Criteria - Arm 1:
Age between 4-65 at enrollment
Clinically affected (defined as weakness on bedside evaluation in either a limb-girdle pattern, or in a distal extremity)
A genetically or functionally confirmed mutation in ANO5, CAPN3, DYSF, DNAJB6 or SGCA-G.
Willing and able to give informed consent and follow all study procedures and requirements
Inclusion Criteria - Arm 2:
Age between 4-65 at enrollment
Clinically affected (defined as weakness on bedside evaluation in either a limb-girdle pattern, or in a distal extremity)
a genetically confirmed mutation in SGCA-G
Willing and able to give informed consent and follow all study procedures and requirements
Exclusion Criteria - Arm 1:
Any other illness that would interfere with the ability to undergo safe testing or would interfere with interpretation of the results in the opinion of the site investigator.
History of a bleeding disorder, platelet count <50,000, current use of an anticoagulant.
Positive pregnancy test at time any timepoint during the trial.
Exclusion Criteria - Arm 2:
Any other illness that would interfere with the ability to undergo safe testing or would interfere with interpretation of the results in the opinion of the site investigator.
History of a bleeding disorder, platelet count <50,000, current use of an anticoagulant
Positive pregnancy test at time any timepoint during the trial.
Rare Diseases
AN05, CAPN3, DNAJB6, DYSF, LGMD, Limb Girdle Muscular Dystrophy, SGCA, SGCB, SGCD, SGCG, Sarcoglycan
Improving Diagnosis and Treatment in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
This is a multi-center, prospective, observational cohort registry study looking at kids and their relatives with Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT).
Bradley Clark
Male or Female
Not specified
STUDY00011733
STUDY00011733
Inclusion Criteria:
• Any patient who has received a diagnosis of CPVT, diagnosed prior to 19 years of age. A diagnosis of CPVT is typically made in the presence of a structurally normal heart, normal QTc interval on resting ECG, and a clinical phenotype of exertional or emotional syncope or cardiac arrest and at least one of the following:
• Unexplained exercise or catecholamine-induced bidirectional VT or polymorphic ventricular premature beats or VT.
• Identification of a heterozygous pathogenic/likely pathogenic variant or biallelic pathogenic/likely pathogenic variants in a gene known to be associated with CPVT OR
• Any patient known to be heterozygous for a pathogenic/likely pathogenic variant or biallelic pathogenic/likely pathogenic variants in a gene known to be associated with CPVT, including those with an atypical phenotype
Exclusion Criteria:
Significant co-morbid condition not directly related to a complication of CPVT. Rare Diseases, Heart & Vascular
CPVT
National Pediatric Cardiology Quality Improvement Collaborative (NPC-QIC) - A Collaborative Initiative to Improve Care of Children with Complex Congenital Heart Disease (NPC-QIC)
Kavisha Shah
All
to 15 Months old
STUDY00004329
STUDY00004329
Inclusion Criteria:
Fetuses or newborns diagnosed with HLHS or other univentricular condition
Intended to undergo Norwood procedure
Exclusion Criteria:
None Collaborative Learning Network, Collaborative Learning Network
Hypoplastic Left Heart Syndrome (HLHS)
PEPN2111 - A Phase 1/2 Trial of CBL0137 (NSC# 825802, IND# 155843) in Patients with Relapsed or Refractory Solid Tumors including CNS Tumors and Lymphoma
A Phase I/II trial of single agent intravenous CBL0137 in pediatric patients (≥ 12 months and ≤ 30 years) with relapsed/refractory solid tumors, including CNS tumors and lymphoma.
Emily Greengard
All
12 Months to 30 Years old
Phase 1/Phase 2
STUDY00015023
STUDY00015023
Inclusion Criteria:
Parts A and B1: Patients must be >= 12 months and =< 21 years of age at the time of study enrollment
Part B2 (relapsed/refractory osteosarcoma): Patients must be >= 12 months and =< 30 years of age at the time of study enrollment
Patients must have had histologic verification of malignancy at original diagnosis or relapse, except in patients with diffuse intrinsic brain stem tumors, or patients with pineal tumors and elevations of cerebrospinal fluid (CSF) or serum tumor markers, including alpha-fetoprotein or beta-human chorionic gonadotropin (HCG)
Part A: Patients with relapsed or refractory solid tumors or lymphoma, including patients with CNS tumors or known CNS metastases (including untreated or progressive) are eligible
Part B1: Patients with progressive or recurrent DIPG (diagnosed by biopsy or imaging characteristics) and other H3 K27M-mutant diffuse midline gliomas previously treated with radiation therapy
Part B2: Patients with relapsed or refractory osteosarcoma
Part A: Patients must have either measurable or evaluable disease
Part B1 and B2: Patients must have measurable disease
Patient's current disease state must be one for which there is no known curative therapy or therapy proven to prolong survival with an acceptable quality of life
Patients must have a performance status corresponding to Easter 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 a Karnofsky or Lansky score >= 50%
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)
Anti-cancer agents not known to be myelosuppressive (eg, 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. Patients with CNS tumors receiving corticosteroids must have been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment
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: >= 30 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
Radiopharmaceutical therapy (e.g., radiolabeled antibody, I-131 metaiodobenzylguanidine [131I MIBG]): >= 42 days after systemically administered radiopharmaceutical therapy
Patients must not have received prior exposure to CBL0137
For patients with solid tumors without known bone marrow involvement:
Peripheral absolute neutrophil count (ANC) >= 1000/uL (performed within 7 days prior to enrollment unless otherwise indicated)
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. 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 must be evaluable for hematologic toxicity
For 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) (performed within 7 days prior to enrollment unless otherwise indicated)
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. 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 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 (performed within 7 days prior to enrollment unless otherwise indicated):
Age: Maximum serum creatinine (mg/dL)
1 to < 2 years: 0.6 (male); 0.6 (female)
2 to < 6 years: 0.8 (male); 0.8 (female)
6 to < 10 years: 1 (male); 1 (female)
10 to < 13 years: 1.2 (male); 1.2 (female)
13 to < 16 years: 1.5 (male); 1.4 (female)
>= 16 years: 1.7 (male); 1.4 (female)
Patients with solid tumors:
Bilirubin (sum of conjugated + unconjugated or total) =< 1.5 x upper limit of normal (ULN) for age (performed within 7 days prior to enrollment unless otherwise indicated)
Patients with solid tumors:
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 (performed within 7 days prior to enrollment unless otherwise indicated)
Shortening fraction of >= 27% by echocardiogram (performed within 7 days prior to enrollment unless otherwise indicated)
Ejection fraction of >= 50% by gated radionuclide study (performed within 7 days prior to enrollment unless otherwise indicated)
Corrected QT (QTC) < 480 msec (performed within 7 days prior to enrollment unless otherwise indicated)
Patients with seizure disorder may be enrolled if seizures well controlled without the use of enzyme-inducing anti-convulsant agents. Well controlled is defined by no increase in seizure frequency in the prior 7 days
Nervous system disorders (Common Terminology Criteria for Adverse Events [CTCAE] version [v]5) resulting from prior therapy must be =< grade 2, with the exception of decreased tendon reflex (DTR). Any grade of DTR is eligible
Patients have consented to receive a central venous catheter prior to the administration of CBL0137. A central line is required for CBL0137 administration
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, OR 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 (except leukemia patients receiving hydroxyurea, which may be continued until 24 hours prior to start of protocol therapy)
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 receiving drugs that are strong inducers or inhibitors of CYP3A4, CYP2B6 (e.g., carbamazepine) and CYP1A2 (e.g., ciprofloxacin, enoxacin, fluvoxamine, smoking) are not eligible. These agents are to be avoided for 7 days prior to the start of CBL0137 and for the duration of the protocol therapy. Sensitive substrates of CYP2D6 (e.g., atomoxetine, desipramine, dextromethorphan, eliglustat, nebivolol, nortriptyline, perphenazine, tolterodine, R-venlafaxine) should also be avoided for the duration protocol therapy
Patients who are receiving drugs that prolong QTc are not eligible. QTc- prolonging drugs are to be avoided for 7 days prior to the start of CBL0137 and for duration of the protocol therapy
Patients with known peripheral vascular disease are excluded
Patients with a history of pro-thrombotic disorder 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 FACT Complex-targeting Curaxin CBL0137, FACT Complex-targeting Curaxin CBL0137
Diffuse Midline Glioma, H3 K27M-Mutant, Metastatic Malignant Neoplasm in the Central Nervous System, Recurrent Diffuse Intrinsic Pontine Glioma, Recurrent Lymphoma, Recurrent Malignant Solid Neoplasm, Recurrent Osteosarcoma, Recurrent Primary Malignant Central Nervous System Neoplasm, Refractory Lymphoma, Refractory Malignant Solid Neoplasm, Refractory Osteosarcoma, Refractory Primary Malignant Central Nervous System Neoplasm
A Randomized Phase III Trial of Adjuvant Therapy Comparing Doxorubicin Plus Cyclophosphamide Followed by Weekly Paclitaxel with or without Carboplatin for Node-Positive or High-Risk Node-Negative Triple-Negative Invasive Breast Cancer
Phase II/III
0123456789
0123456789
Gliosarcoma, MGMT-Unmethylated Glioblastoma
A Randomized Phase III Trial of Adjuvant Therapy Comparing Doxorubicin Plus Cyclophosphamide Followed by Weekly Paclitaxel with or without Carboplatin for Node-Positive or High-Risk Node-Negative Triple-Negative Invasive Breast Cancer
Phase III
0123456789
0123456789
Breast Adenocarcinoma, Estrogen Receptor Negative, HER2/Neu Negative, Progesterone Receptor Negative, Stage IB Breast Cancer, Stage IIA Breast Cancer, Stage IIB Breast Cancer, Stage IIIA Breast Cancer, Stage IIIC Breast Cancer, Triple-Negative Breast Carcinoma
MT2014-10C : Allogeneic Hematopoietic Stem Cell Transplant for Patients with High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders
Ashish Gupta
All
to 55 Years old
N/A
1407M52125
1407M52125
Inclusion Criteria:
Diagnosis of Sickle Cell Disease, Thalassemia, Diamond Blackfan Anemia or other non-malignant hematologic disorders for which a stem cell transplant is indicated
Acceptable stem cell source identified
Performance status of ≥ 70% (Karnofsky),or ≥ 70 (Lansky play score)
Creatinine <2.0 mg/dl for adults or glomerular filtration rate > 50 ml/min for children
Bilirubin, Aspartate Aminotransferase, Alkaline phosphatase <5 times the upper limit of institutional normal
Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction > 40%
Exclusion Criteria:
active, uncontrolled infection
pregnant or breastfeeding
HIV positive Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen, Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen
Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria, Glanzmann Thrombasthenia, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Non-Malignant Hematologic Disorders
Clinics and Surgery Center (CSC)
Targeting Family Meal Quality and Quantity to Reduce Childhood Obesity Using Ecological Momentary Intervention (EMI) and Video Feedback
The proposed study is an individual three-arm randomized controlled tiled aimed at utilizing state-of-the-art intervention methods to examine whether increasing the quality and the quantity of family meals reduces childhood obesity.
Jerica Berge
All
5 Years to 10 Years old
NA
STUDY00000706
STUDY00000706
STUDY INCLUSION CRITERIA
The inclusionary criteria used to select participants for the proposed study includes:
Boys and girls (n=525) ages 5-10 years old who attend a University of Minnesota Physicians (n=4) or Fairview (n=8) primary care clinic and their primary caregiver (e.g., mother, father, grandparent) and at least one sibling. A second primary caregiver and other family members can also participate.
Must eat ≤3 family dinner meals per week. Research shows that youth who have more than three family meals per week are less likely to be obese ten years later. Thus, families who report three or fewer family meals per week will be recruited to optimize the potential for change in family meal frequency. Families will focus on the family dinner in the intervention to improve their family meal quality and quantity. This decision is based on: (1) Family Systems Theory, which indicates that change in one setting (e.g., family dinner) will generalize to other settings (e.g., breakfast, lunch, snacks).
Child with age and sex adjusted BMI ≥75th percentile (no upper limit).
One of the following race/ethnicities: African American, Hispanic/Latino, Native American, Asian American, or White. In order to examine racial/ethnic differences in study hypotheses, equal numbers of children per racial/ethnic group (total=525) will be recruited.
Parent and family members who speak English or Spanish.
Not expected to move within the next two years.
STUDY EXCLUSION CRITERIA
The exclusionary criteria used to exclude participants from the proposed study includes:
Children with medically necessary dietary restrictions (reviewed by MD from primary care clinics).
Non-custodial parents, who the child does not live with more than 50% of the time.
In-home Visits and Food Preparation Activities, Ecological Momentary Intervention, Feedback on Video-recorded Family Meals, Maintenance, In-home Visits and Food Preparation Activities, Ecological Momentary Intervention, Feedback on Video-recorded Family Meals, Maintenance
Childhood Obesity
Childhood Obesity, Ecological Momentary Intervention, Video Feedback
ZEUS - Effects of ziltivekimab versus placebo on cardiovascular outcomes in patients with established atherosclerotic cardiovascular disease, chronic kidney disease and systemic inflammation (ZEUS)
We are doing this study to see if ziltivekimab reduces the risk of having cardiovascular events (for example heart attack and stroke) in people with cardiovascular disease, chronic kidney disease and inflammation. Participants will either get ziltivekimab (active medicine) or placebo (a dummy medicine which has no effect on the body). This is known as the study medicine. Which treatment participants get is decided by chance. Participants chance of getting ziltivekimab or placebo is the same. Ziltivekimab is not yet approved in any country or region in the world. It is a new medicine doctors cannot prescribe. Participants will get the study medicine in a pre filled syringe. Participants will need to use the pre filled syringe to inject the study medicine into a skinfold once-monthly. The study is expected to last for 2.5 to 4 years. Participants will have up to 20 clinic visits. Participants will have blood and urine samples taken at most of the clinic visits. Participants will have their heart examined using sound waves (echocardiography) and electrodes (electrocardiogram). Women cannot take part if pregnant, breast-feeding or planning to get pregnant during the study period.
Daniel Duprez
All
18 Years to old
Phase III
STUDY00013843
STUDY00013843
Inclusion Criteria:
Chronic kidney disease defined by one of the below:
Estimated glomerular filtration rate (eGFR) greater than or equal to (>=) 15 and below 60 mL/min/1.73 m^2 (using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation)
Urinary albumin-to-creatinine ratio (UACR) >= 200 milligrams per gram (mg/g) and eGFR >= 60 mL/min/1.73 m2 (using the CKD-EPI creatinine equation)
Serum high-sensitivity C-reactive protein (hs-CRP) greater than or equal to 2 milligram per liter (mg/L)
Evidence of atherosclerotic cardiovascular disease (ASCVD) by one or more of the following:
a) Coronary heart disease defined as at least one of the following: i. Documented history of MI ii. Prior coronary revascularisation procedure iii. greater than or equal to 50% stenosis in major epicardial coronary artery documented by cardiac catheterisation or CT coronary angiography b) Cerebrovascular disease defined as at least one of the following: i. Prior stroke of atherosclerotic origin ii. Prior carotid artery revascularisation procedure iii. greater than or equal to 50% stenosis in carotid artery documented by X-ray angiography, MR angiography, CT angiography or Doppler ultrasound.
c) Symptomatic peripheral artery disease (PAD) defined as at least one of the following: i. Intermittent claudication with an ankle-brachial index (ABI) below or equal to 0.90 at rest ii. Intermittent claudication with a greater than or equal to 50% stenosis in peripheral artery (excluding carotid) documented by X-ray angiography, MR angiography, CT angiography or Doppler ultrasound iii. Prior peripheral artery (excluding carotid) revascularisation procedure iv. Lower extremity amputation at or above ankle due to atherosclerotic disease (excluding e.g. trauma or osteomyelitis).
Exclusion Criteria:
Clinical evidence of, or suspicion of, active infection at the discretion of the investigator.
Myocardial infarction, stroke, hospitalisation for unstable angina pectoris, or transient ischaemic attack within 60 days prior to randomisation (visit 2).
Planned coronary, carotid or peripheral artery revascularisation known on the day of randomisation (visit 2).
Major cardiac surgical, non-cardiac surgical, or major endoscopic procedure (thoracoscopic or laparoscopic) within the past 60 days prior to randomisation (visit 2) or any major surgical procedure planned at the time of randomisation (visit 2). Ziltivekimab, Placebo (ziltivekimab), Ziltivekimab, Placebo (ziltivekimab)
Cardiovascular Risk, Chronic Kidney Disease, Inflammation
Clinics and Surgery Center (CSC)
Transcatheter Replacement of Stenotic Aortic Valve through Implantation of ACURATE in Subjects InDicatEd for TAVR
Mudassar Ahmed
All
Pivotal
STUDY00007377
STUDY00007377
Inclusion Criteria:
IC1. Subject has documented severe symptomatic native aortic stenosis defined as follows: aortic valve area (AVA) ≤1.0 cm2 (or AVA index ≤0.6 cm2/m2) AND a mean pressure gradient ≥40 mmHg, OR maximal aortic valve velocity ≥4.0 m/s, OR Doppler velocity index ≤0.25 as measured by echocardiography and/or invasive hemodynamics.
Note: In cases of low flow, low gradient aortic stenosis with left ventricular dysfunction (ejection fraction <50%), dobutamine can be used to assess the grade of aortic stenosis (maximum dobutamine dose of 20 mcg/kg/min recommended); the subject may be enrolled if echocardiographic criteria are met with this augmentation.
IC2. Subject has a documented aortic annulus size of ≥21 mm and ≤29 mm based on the center's assessment of pre-procedure diagnostic imaging (and confirmed by the Case Review Committee [CRC]) and, for the randomized cohort, is deemed treatable with an available size of both test and control device.
IC3. For subjects with symptomatic aortic valve stenosis per IC1 definition above, functional status is NYHA Functional Class ≥ II.
IC4. Heart team (which must include an experienced cardiac interventionalist and an experienced cardiac surgeon) agrees that the subject is indicated for TAVR, is likely to benefit from valve replacement, and TAVR is appropriate.
IC5. Subject (or legal representative) understands the study requirements and the treatment procedures, and provides written informed consent.
IC6. Subject, family member, and/or legal representative agree(s) and subject is capable of returning to the study hospital for all required scheduled follow up visits.
IC7. Subject is expected to be able to take the protocol-required adjunctive pharmacologic therapy.
Exclusion Criteria:
EC1. Subject has a unicuspid or bicuspid aortic valve.
EC2. Subject has had an acute myocardial infarction within 30 days prior to the index procedure (defined as Q-wave MI or non-Q-wave MI with total CK elevation ≥ twice normal in the presence of CK-MB elevation and/or troponin elevation).
EC3. Subject has had a cerebrovascular accident or transient ischemic attack clinically confirmed by a neurologist or neuroimaging within the past 6 months prior to study enrollment.
EC4. Subject is on renal replacement therapy or has eGFR <20.
EC5. Subject has a pre-existing prosthetic aortic or mitral valve.
EC6. Subject has severe (4+) aortic, tricuspid, or mitral regurgitation.
EC7. Subject has moderate or severe mitral stenosis (mitral valve area ≤1.5 cm2 and diastolic pressure half-time ≥150 ms, Stage C or D76).
EC8. Subject has a need for emergency surgery for any reason.
EC9. Subject has a history of endocarditis within 6 months of index procedure or evidence of an active systemic infection or sepsis.
EC10. Subject has echocardiographic evidence of new intra-cardiac vegetation or intraventricular or paravalvular thrombus requiring intervention.
EC11. Subject has platelet count <50,000 cells/mm3 or >700,000 cells/mm3, or white blood cell count <1,000 cells/mm3.
EC12. Subject has had a gastrointestinal bleed requiring hospitalization or transfusion within the past 3 months, or has other clinically significant bleeding diathesis or coagulopathy that would preclude treatment with required antiplatelet regimen, or will refuse transfusions.
EC13. Subject has known hypersensitivity to contrast agents that cannot be adequately pre-medicated, or has known hypersensitivity to the protocol required medications (aspirin, all P2Y12 inhibitors, heparin), or to the individual components of the test or control valve (nickel, titanium, stainless steel, platinum, iridium or polyethylene terephthalate [PET]).
EC14. Subject has a life expectancy of less than 12 months due to non-cardiac, comorbid conditions based on the assessment of the investigator at the time of enrollment.
EC15. Subject has hypertrophic cardiomyopathy.
EC16. Subject has any therapeutic invasive cardiac or vascular procedure within 30 days prior to the index procedure (except for balloon aortic valvuloplasty, pacemaker implantation, or implantable cardioverter defibrillator implantation, which are allowed).
EC17. Subject has untreated coronary artery disease, which in the opinion of the treating physician is clinically significant and requires revascularization.
EC18. Subject has severe left ventricular dysfunction with ejection fraction <20%.
EC19. Subject is in cardiogenic shock or has hemodynamic instability requiring inotropic support or mechanical support devices.
EC20. Subject has arterial access that is not acceptable for the study device (test or control) delivery systems as defined in the device (test or control) Directions For Use.
EC21. Subject has either of the following:
Severe vascular disease that would preclude safe access (e.g., aneurysm with thrombus that cannot be crossed safely; marked tortuosity; significant narrowing of the abdominal aorta; severe unfolding of the thoracic aorta; or thick, protruding, ulcerated atheroma in the aortic arch), OR
Severe/eccentric calcification of the aortic annulus that would prevent safe implantation of the TAVR prosthesis.
EC22. Subject has current problems with substance abuse (e.g., alcohol, etc.) that may interfere with the subject's participation in this study.
EC23. Subject is participating in another investigational drug or device study that has not reached its primary endpoint or subject intends to participate in another investigational device clinical trial within 12 months after index procedure.
EC24. Subject has untreated conduction system disorder (e.g., Type II second degree atrioventricular block) that in the opinion of the treating physician is clinically significant and requires a pacemaker implantation. Enrollment is permissible after permanent pacemaker implantation.
EC25. Subject has severe incapacitating dementia.
Additional exclusion criteria apply to subjects considered for enrollment in the CT Imaging Substudy as listed below.
AEC1. Subject has eGFR <30 mL/min (chronic kidney disease stage IV or stage V)
AEC2. Subject has atrial fibrillation that cannot be rate controlled to ventricular response rate < 60 bpm.
AEC3. Subject is expected to undergo chronic anticoagulation therapy after the index procedure.
Note: Subjects treated with short-term anticoagulation post procedure can be included in the CT Imaging Substudy; in these subjects the 30-day imaging will be performed 30 days after discontinuation of anticoagulation. ACURATE neo2™ Transfemoral TAVR System, Medtronic CoreValve TAVR System, Edwards SAPIEN 3 TAVR System, ACURATE Prime™ Transfemoral TAVR System XL, ACURATE neo2™ Transfemoral TAVR System, Medtronic CoreValve TAVR System, Edwards SAPIEN 3 TAVR System, ACURATE Prime™ Transfemoral TAVR System XL
Aortic Stenosis
Type 1 Diabetes Extension Study (T1DES)
The primary objective of the study will be to further our understanding of the immunologic mechanisms underlying maintenance and loss of beta cell function by evaluating the relationship between longitudinal changes in beta cell function and changes over time in biomarkers known to be associated with a response to immune modulating treatments. This is meant to be a follow up study of the long term effects of participation in selected completed ITN new-onset T1D studies with immunomodulatory agents.
Antoinette Moran
All
8 Years to 35 Years old
STUDY00001310
STUDY00001310
Inclusion Criteria:
Prior participant in an Immune Tolerance Network (ITN) executive committee approved T1DM study.
Ability to sign informed consent/assent (as applicable for children).
Exclusion Criteria:
Any medical condition that in the opinion of the principal investigator would interfere with safe completion of the trial; or
Inability to comply with the study visit schedule and required assessments. Type 1 Diabetes Mellitus, T1DM, T1D
Insulin, Glucose Intolerance
Maternal Metabolism, Breastmilk Composition, and Transmission to Infants
This is a pilot prospective cohort study of the differences between women with and without diabetes during pregnancy in their breast milk composition (microbiome and hormone composition), and test for group differences in the relationship of breast milk composition to infant gut microbiome characteristics, weight gain, and body composition. Women with diabetes during pregnancy (N=50) will be recruited de novo in this study, while women without diabetes (N=100) already have been enrolled and have provided consent for all necessary data involved in the comparison with the diabetic women, except that the non-diabetic women have not provided consent for the meta-genomic sequencing analysis and so will provide that consent under this protocol.
Ellen Demerath
All
STUDY00002127
STUDY00002127
Inclusion Criteria:
(For new enrollment of 50 pregnant women with gestational diabetes and their infants. Enrollment of normal weight and obese cohort comparators already accomplished under ClinicalTrial NCT03301753):
Pregnant women
age 21-45 at time of delivery
report during enrollment procedures that they have social support for and intention to exclusively breastfeed for at least 3 months (breastfeeding intentions are known to be correlated with actual behavior), and if parity >1, that they successfully breastfed after a previous pregnancy for at least 3 months
singleton pregnancy
known gestational diabetes Definition of Gestational Diabetes: 1) an elevated glucose challenge test >200 mg/dL or 2) two abnormal values on the glucose tolerance test according to Carpenter-Coustan criteria.
Exclusion Criteria:
alcohol consumption >1 drink per week during pregnancy/lactation
tobacco consumption during pregnancy/lactation,
inability to speak/understand English
known congenital metabolic, endocrine disease, or congenital illness affecting infant feeding
planned delivery at a site other than the University of Minnesota Medical Center- West Bank campus.
preexisting diabetes Obesity, Diabetes, Gestational
Obesity, Gestational Diabetes, Breast Milk, Microbiome, Infant Growth
MT2012-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
Christen Ebens
All
to 50 Years old
N/A
1207M17321
1207M17321
Inclusion Criteria:
Diagnosis of immunodeficiency or histiocytic disorder including the following:
Severe combined immunodeficiency (SCID - all variants)
Second bone marrow transplant (BMT) for SCID (after graft rejection)
Omenn's Syndrome
Reticular dysgenesis
Wiskott-Aldrich syndrome
Major histocompatibility complex (MHC) Class II deficiency (bare lymphocyte syndrome)
Hyper IgM Syndrome (CD40 Ligand Deficiency)
Common variable immunodeficiency (CVID) with severe phenotype
Chronic Granulomatous Disease (CGD)
Other severe Combined Immune Deficiencies (CID)
Hemophagocytic Lymphohistiocytosis (HLH)
X-linked Lymphoproliferative Disease (XLP)
Chediak-Higashi Syndrome (CHS)
Griscelli Syndrome
Langerhans Cell Histiocytosis (LCH)
Acceptable stem cell sources include:
HLA identical or 1 antigen matched sibling donor eligible to donate bone marrow
HLA identical or up to a 1 antigen mismatched unrelated BM donor
Sibling donor cord blood with acceptable HLA match and cell dose as per current institutional standards
Single unrelated umbilical cord blood unit with 0-2 antigen mismatch and minimum cell dose of >5 x 10^7 nucleated cells/kg as per current institutional guidelines
Double unrelated umbilical cord blood units that are:
up to 2 antigen mismatched to the patient
up to 2 antigen mismatched to each other
minimum cell dose of at least one single unit must be ≥ 3.5 x 10^7 nucleated cells/kg
combined dose of both units must provide a total cell dose of ≥ 5 x 10^7 nucleated cells/kg
Age: 0 to 50 years
Adequate organ function and performance status.
Exclusion Criteria
pregnant or breastfeeding
active, uncontrolled infection and/or HIV positive
acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy Alemtuzumab 0.3 mg, Cyclophosphamide, Busulfan, Stem Cell Transplantation, Fludarabine phosphate 40 mg, Melphalan, Alemtuzumab 0.2 mg, Busulfan, Fludarabine phosphate 30 mg, MESNA, Alemtuzumab 0.3 mg, Cyclophosphamide, Busulfan, Stem Cell Transplantation, Fludarabine phosphate 40 mg, Melphalan, Alemtuzumab 0.2 mg, Busulfan, Fludarabine phosphate 30 mg, MESNA
SCID, Omenn's Syndrome, Reticular Dysgenesis, Wiskott-Aldrich Syndrome, Bare Lymphocyte Syndrome, Common Variable Immunodeficiency, Chronic Granulomatous Disease, CD40 Ligand Deficiency, Hyper IgM Syndrome, X-linked Lymphoproliferative Disease, Hemophagocytic Lymphohistiocytosis, Griscelli Syndrome, Chediak-Higashi Syndrome, Langerhan's Cell Histiocytosis
Clinics and Surgery Center (CSC)
Phase 1/2 Study to Evaluate Palbociclib (IBRANCE?) in Combination With Irinotecan and Temozolomide or in Combination with Topotecan and Cyclophosphamide in Pediatric Patients With Recurrent or Refractory Solid Tumors Protocol No.: ADVL1921/A5481092
This is a Phase 1/2 multicenter, open-label study to evaluate palbociclib in combination with either irinotecan (IRN) and temozolomide (TMZ) or topotecan (TOPO) and cyclophosphamide (CTX) chemotherapy in children, adolescents and young adults with recurrent or refractory solid tumors. The study consists of a non- randomized Phase 1 portion for recurrent or refractory solid tumors followed by potential non- randomized tumor specific cohort(s) and a randomized, Phase 2 portion for recurrent or refractory EWS.
Emily Greengard
All
2 Years to 20 Years old
Phase 2
STUDY00007068
STUDY00007068
Inclusion:
Histologically confirmed relapsed or refractory solid tumor as follows:
For dose escalation and dose determination parts: Histologically confirmed relapsed or refractory solid tumor (including CNS tumors but not lymphomas). Patients with Diffuse Intrinsic Pontine Glioma do not require histological only radiographic confirmed relapse to enroll.
For dose expansion and tumor specific cohorts: Histologically confirmed relapsed or refractory solid tumor including but not limited to EWS, rhabdoid tumor, rhabdomyosarcoma, neuroblastoma, and medulloblastoma. Patients with Diffuse Intrinsic Pontine Glioma do not require histological only radiographic confirmed relapse to enroll. EWS is not eligible for TOPO and CTX tumor-specific cohorts.
For randomized Phase 2 part: Histologically confirmed Ewing sarcoma at diagnosis or at relapse, with presence of EWSR1-ETS or FUS-ETS rearrangement. Histopathology confirmation of both EWSR1-ETS or FUS-ETS rearrangement partners is required OR availability of formalin fixed paraffin embedded (FFPE) tumor tissue sample for central testing. Patient must have relapsed or have refractory disease and at least evaluable disease in at least one site other than bone marrow that can be followed by imaging.
Age ≥2 and <21 years at the time of study entry.
Lansky performance status ≥50% for patients ≤16 years of age, or Eastern Cooperative Oncology Group (ECOG) 0, 1 or 2 for patients >16 years of age.
Adequate bone marrow function.
Absolute neutrophil count ≥1000/mm3;
Platelet count ≥100,000/mm3 (transfusion independent, no platelet transfusion in past 7 days prior study entry);
Hemoglobin ≥8.5 g/dL (transfusion allowed).
Adequate renal function: Serum creatinine level based on age/gender must within protocol specified limits.
Adequate liver function, including:
Aspartate aminotransferase (AST) and alanine aminotransferase (ALT)
≤2.5 × upper limit of normal (ULN) or ≤5 × ULN for age, if attributable to disease involvement of the liver;
Total bilirubin ≤1.5 × ULN for age, unless the patient has documented Gilbert's syndrome.
Patients enrolled to Phase 1 portion of the study and tumor specific cohorts must have measurable disease as defined by RECIST version 1.1 or modified RANO criteria for CNS disease or INRC for neuroblastoma. Patients with EWS enrolled to Phase 2 portion of the study are eligible with evaluable disease (eg, bone only disease with no soft tissue component).
Recovered to CTCAE Grade ≤1, or to baseline, from any non-hematological acute toxicities of prior surgery, chemotherapy, immunotherapy, radiotherapy, differentiation therapy or biologic therapy, with the exception of alopecia.
Serum/urine pregnancy test (for all girls ≥8 years of age) negative at screening and at the baseline visit.
Exclusion:
Phase 1 and tumor specific cohorts: For palbociclib with IRN and TMZ combination, prior treatment with a CDK4/6 inhibitor or progression while on treatment with an IRN-containing regimen that includes TMZ. Patients who have received the combination of IRN and TMZ and did not progress while on these medications are eligible. For patients enrolling in the palbociclib with TOPO and CTX combination, prior treatment with a CDK4/6 inhibitor or progression while on treatment with a TOPO-containing regimen that includes CTX. Patients who have received the combination of TOPO and CTX and did not progress while on these medications are eligible. Phase 2 portion: prior treatment with a CDK4/6 inhibitor or progression while on treatment with an IRN-containing or TMZ-containing regimen. Patients who have received IRN and/or TMZ and did not progress while on these medications are eligible.
Prior intolerability to IRN and/or TMZ plus/minus palbociclib with IRN and TMZ combination and prior intolerability to TOPO and/or CTX for TOPO and CTX combination.
Use of strong cytochrome P450 (CYP) 3A inhibitors or inducers. Patients who are receiving strong uridine diphosphate-glucuronosyl transferase 1A1 (UGT1A1) inhibitors within 12 days of Cycle 1 Day 1 (C1D1) are not eligible for the palbociclib with IRN and TMZ combination. Patients who are receiving strong UGT1A1 inhibitors within 12 days of C1D1 are eligible for the palbociclib with TOPO and CTX combination (See Section 5.7.1 for list of products.)
Systemic anti cancer therapy within 2 weeks prior to study entry and 6 weeks for nitrosoureas.
Prior irradiation to >50% of the bone marrow (see Appendix 9).
Participation in other studies involving investigational drug(s) within 2 weeks or 5 half lives, whichever is longer, prior to study entry.
Major surgery within 4 weeks prior to study entry. Surgical biopsies or central line placement are not considered major surgeries.
For IRN and TMZ with/without palbociclib combinations: known or suspected hypersensitivity to palbociclib, IRN and/or TMZ. For combination of palbociclib with TOPO and CTX: known or suspected hypersensitivity to palbociclib, TOPO and/or CTX.
Patients with known symptomatic brain tumors or brain metastases and require steroids, unless they have been on a stable or on a decreasing steroid dose for >14 days.
Patients with previously diagnosed brain metastases are eligible if they have completed their prior treatment and have recovered from the acute effects of radiation therapy or surgery prior to study entry for these metastases for at least 14 days post radiation and 4 weeks post-surgery and are neurologically stable.
Hereditary bone marrow failure disorder.
QTc >470 msec.
History of clinically significant or uncontrolled cardiac disease, including:
History of or active congestive heart failure; if patient had congestive heart failure resolve and >1 year from resolution, patient will be considered eligible;
Clinically significant ventricular arrhythmia (such as ventricular tachycardia, ventricular fibrillation or Torsades de Pointes);
Diagnosed or suspected congenital or acquired prolonged QT syndrome;
Need for medications known to prolong the QT interval;
Uncorrected hypomagnesemia or hypokalemia because of potential effects on the QT interval;
Left ventricular ejection fraction <50% or shortening fraction <28%.
Recent or ongoing clinically significant gastrointestinal disorder that may interfere with absorption of orally administered drugs (eg, gastrectomy).
Severe acute or chronic medical or laboratory test abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results, and in the judgment of the Investigator, would make the patient inappropriate for entry into this study.
Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or patients who are Pfizer employees, including their family members, directly involved in the conduct of the study.
Palbociclib, Temozolomide, Irinotecan, Topotecan, Cyclophosphamide, Palbociclib, Temozolomide, Irinotecan, Topotecan, Cyclophosphamide
Ewing Sarcoma, Solid Tumors, Rhabdoid Tumor, Rhabdomyosarcoma, Neuroblastoma, Medulloblastoma, Diffuse Intrinsic Pontine Glioma
Ewing Sarcoma, EWS, Solid Tumor, Recurrent Solid Tumors, Refractory Solid Tumors, Bone Cancer, Bone Tumor, Bone Sarcoma, Soft Tissue Cancer, Soft Tissue Sarcoma, Recurrent Ewing Sarcoma, Refractory Ewing Sarcoma, Relapsed Ewing Sarcoma, Pediatric Cancer, Childhood Cancer, Ewing Sarcoma Treatment, Palbociclib, CDK4/6 Inhibitor, Irinotecan, Temozolomide, Topotecan, Cyclophosphamide
Corrona Inflammatory Bowel Disease (IBD) Registry
This is a prospective, non-interventional, research study for patients with IBD under the care of a certified gastroenterologist. The primary objective for this registry is to prospectively study the natural history of IBD, the prevalence and incidence of comorbidities, targeted adverse events, and more, via questionnaires.
Byron Vaughn
All
18 Years to old
STUDY00007736
STUDY00007736
ELIGIBILITY CRITERIA∗ To be eligible for enrollment into the Corrona IBD Registry, a patient must satisfy all of the inclusion criteria and none of the exclusion criteria listed below.
Inclusion Criteria:
At least 18 years of age or older.
Willing and able to provide written consent for participation in the IBD Registry.
Willing and able to provide Personally Identifiable Information (PII) which includes the following types of personal information at a minimum: full name, date of birth, sex, and home address zip code.
Diagnosis of one of the following by a gastroenterologist:
Crohn's disease
Ulcerative colitis
Prevalent users or new /incident users of an approved biologic drug or JAK inhibitor (Tofacitinib) for the treatment of UC or Crohn's disease.
Exclusion Criteria:
• Participating in or planning to participate in a clinical trial (Phase I - III) or a post-marketing study or registry (i.e. phase IV).∆
Eligible Medications Grouped by Drug Class
ANTI-TNF AGENTS AND BIOSIMILARS - Adalimumab (HUMIRA), Adalimumab-atto (AMJEVITA), Certolizumab pegol (CIMZIA), Golimumab (SIMPONI), Infliximab (REMICADE), Infliximab-dyyb (INFLECTRA)
INTEGRIN RECEPTOR ANTAGONISTS - Natalizumab (TYSABRI), Vedolizumab (ENTYVIO)
INTERLEUKIN ANTAGONIST (IL-12 AND IL-23), Ustekinumab (STELARA),
JAK INHIBITOR - Tofacitinib (XELJANZ)
SPHINGSOSINE-1-PHOSPHATE RECEPTOR (S1PR) - Ozanimod (ZEPOSIA)
∆ Once clinical trial participation has ended, a patient is permitted to enroll in the registry if they satisfy the eligibility requirements.
∗ These criteria are subject to change with the needs of the registry at the sole discretion of the Sponsor (Corrona). Inflammatory Bowel Diseases
Clinics and Surgery Center (CSC)
PRI-VENT FSGS: Preemptive Rituximab to Prevent Recurrent Focal Segmental Glomerulosclerosis Post-Transplant
PRI-VENT FSGS is a phase III, multicenter, randomized, open label, clinical trial to test the hypothesis that plasmapheresis plus rituximab prior to kidney transplantation can prevent recurrent FSGS in children and adults.
Michelle Rheault
All
1 Year to 65 Years old
Early Phase 1
STUDY00004388
STUDY00004388
In order to be eligible to participate in this study, an individual must meet all of the following criteria:
Age 1-65 years at the time of kidney transplant
Biopsy proven diagnosis of primary FSGS or minimal change disease
History of nephrotic syndrome (proteinuria, edema, hypoalbuminemia)
First kidney transplant or second or third transplant with a history of recurrent FSGS in the first or second kidney transplant.
The patient (if ≥18 years old) or the child's parent or guardian must be able and willing to give written informed consent and comply with the requirements of the study protocol. Patient assent if <18 years old will be required per local IRB requirements.
Negative urine pregnancy test prior to randomization (for females who are post-menarche).
Males and females of reproductive potential (sexually active in boys or post-menarche in girls) must agree to use an acceptable method of birth control during treatment and for twelve months (1 year) after completion of treatment with rituximab.
An individual who meets any of the following criteria will be excluded from participation in this study:
• Known genetic cause of FSGS 2. Patients with FSGS secondary to another condition (obesity, viral infection, medications, etc.) 3. 4. Received rituximab within 1 year prior to transplant 5. Known hypersensitivity to rituximab, to any of its excipients, or to murine proteins 6. History of severe allergic or anaphylactic reactions to humanized or murine monoclonal antibodies 7. Known active bacterial, viral (e.g. HIV, hepatitis B, hepatitis C), fungal, mycobacterial, or other infection (including tuberculosis or atypical mycobacterial disease, but excluding fungal infections of nail beds) or any major episode of infection requiring hospitalization or treatment with iv antibiotics within 4 weeks of screening or oral antibiotics within 2 weeks prior to screening visit.
• Participation in another therapeutic trial within 30 days of enrollment or 5 half-lives of the investigational drug (whichever is longer) 9. ANC < 1.5 x 103 10. Hemoglobin: < 8.0 gm/dL 11. Platelets: < 100,000/mm 12. AST or ALT >2.5 x Upper Limit of Normal at the local institution's laboratory 13. History of drug, alcohol, or chemical abuse within 6 months prior to screening visit.
• Pregnant, lactating, or refusal of birth control in an adolescent of child-bearing potential 15. Concomitant malignancies or previous malignancies 16. History of psychiatric disorder that would interfere with normal participation in this protocol 17. History of significant cardiac (including arrhythmias) or pulmonary disease (including obstructive pulmonary disease) 18. Any other disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates the use of an investigational drug or that may affect the interpretation of the results or render the patient at high risk from treatment complications 19. Inability to comply with study and follow-up procedures
• Known genetic cause of FSGS 2. Patients with FSGS secondary to another condition (obesity, viral infection, medications, etc.) 3. 4. Received rituximab within 1 year prior to transplant 5. Known hypersensitivity to rituximab, to any of its excipients, or to murine proteins 6. History of severe allergic or anaphylactic reactions to humanized or murine monoclonal antibodies 7. Known active bacterial, viral (e.g. HIV, hepatitis B, hepatitis C), fungal, mycobacterial, or other infection (including tuberculosis or atypical mycobacterial disease, but excluding fungal infections of nail beds) or any major episode of infection requiring hospitalization or treatment with iv antibiotics within 4 weeks of screening or oral antibiotics within 2 weeks prior to screening visit.
• Participation in another therapeutic trial within 30 days of enrollment or 5 half-lives of the investigational drug (whichever is longer) 9. ANC < 1.5 x 103 10. Hemoglobin: < 8.0 gm/dL 11. Platelets: < 100,000/mm 12. AST or ALT >2.5 x Upper Limit of Normal at the local institution's laboratory 13. History of drug, alcohol, or chemical abuse within 6 months prior to screening visit.
• Pregnant, lactating, or refusal of birth control in an adolescent of child-bearing potential 15. Concomitant malignancies or previous malignancies 16. History of psychiatric disorder that would interfere with normal participation in this protocol 17. History of significant cardiac (including arrhythmias) or pulmonary disease (including obstructive pulmonary disease) 18. Any other disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates the use of an investigational drug or that may affect the interpretation of the results or render the patient at high risk from treatment complications 19. Inability to comply with study and follow-up procedures
Rituximab, Plasmapheresis, Rituximab, Plasmapheresis
Focal Segmental Glomerulosclerosis
Clinics and Surgery Center (CSC)
Inflammatory Neuropathy Consortium data Base (INCBase)
INCBase is a prospective, international Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) registry with the primary objective of developing a prognostic model that predicts long-term treatment response in CIDP patients at the start of treatment.
Jeff Allen
Male or Female
Not specified
STUDY00010626
STUDY00010626
Inclusion Criteria:
A clinical diagnosis of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), as determined by the treating physician
Exclusion Criteria:
Any condition that limits the participants ability to
provide accurate study information in a timely and reliable manner Rare Diseases
Clinics and Surgery Center (CSC), Chronic Inflammatory Demyelinating Polyneuropathy, Rare Diseases, Inflammatory Neuropathy
A Phase 1b/2 Study of Abemaciclib in Combination with Irinotecan and Temozolomide (Part A) and Abemaciclib in Combination with Temozolomide (Part B) in Pediatric and Young Adult Patients with Relapsed/Refractory Solid Tumors and Abemaciclib in Combination with Dinutuximab, GM-CSF, Irinotecan, and Temozolomide in Pediatric and Young Adult Patients with Relapsed/Refractory Neuroblastoma (Part C). Protocol Number: I3Y-MC-JPCS
The study's purpose is to see if the drug abemaciclib is safe and effective in combination with temozolomide and irinotecan (Part A) and abemaciclib in combination with temozolomide (Part B) in pediatric and young adult participants with relapsed/refractory solid tumors.
Emily Greengard
All
to 21 Years old
Phase 1/Phase 2
STUDY00013998
STUDY00013998
Inclusion Criteria:
Parts A and B only:
Participants must be less than or equal to (≤)18 years of age.
Body weight greater than or equal to (≥)10 kilograms and body surface area (BSA) ≥0.5 -- Participants with any relapsed/refractory malignant solid tumor (excluding lymphoma), including central nervous system tumors, that have progressed on standard therapies.
For sites that are actively enrolling Parts B and C, participants with neuroblastoma who are eligible for Part C will be excluded from Part B unless approved by Lilly CRP/CRS.
Part C only:
Participants must be less than (<) 21 years of age.
Participants have a BSA ≥0.3 m².
Participants with first relapse/refractory neuroblastoma.
All Parts
Participants must have measurable or evaluable disease by RECIST v1.1 or RANO.
A Lansky score ≥50 for participants <16 years of age or Karnofsky score ≥50 for participants ≥16 years of age.
Participants must have discontinued all previous treatments for cancer or investigational agents and must have recovered from the acute effects to Grade ≤1 at the time of enrollment.
Able to swallow.
Adequate hematologic and organ function ≤2 weeks (14 days) prior to first dose of study drug.
Females of reproductive potential must have negative urine or serum pregnancy test at baseline (within 7 days prior to starting treatment).
Female participants of reproductive potential must agree to use highly effective contraceptive precautions during the trial. For abemaciclib, females should use contraception for at least 3 weeks following the last abemaciclib. For other study drugs, highly effective contraceptive precautions (and avoiding sperm donation) must be used according to their label.
Life expectancy of at least 8 weeks and able to complete at least 1 cycle of treatment.
Caregivers and participants willing to make themselves available for the duration of the trial.
Exclusion Criteria:
Received allogenic bone marrow or solid organ transplant.
Received live vaccination.
Intolerability or hypersensitivity to any of the study treatments or its components.
Diagnosed and/or treated additional malignancy within 3 years prior to enrollment that may affect the interpretation of results, with the exception of curatively treated basal cell carcinoma of the skin, squamous cell carcinoma of the skin, and/or curatively resected in situ cervical and/or breast cancers.
Pregnant or breastfeeding.
Active systemic infections or viral load.
Serious and/or uncontrolled preexisting medical condition(s) that would preclude participation in this study.
Parts A and C only: Have a bowel obstruction.
Prior treatment with drugs known to be strong inhibitors or inducers of isoenzyme cytochrome P450 3A (CYP3A) or strong inhibitors of uridine diphosphate-glucuronosyl transferase 1A1 (UGT1A1) if the treatment cannot be discontinued or switched to a different medication at least 5 half-lives prior to starting study drug.
Received prior treatment with cyclin-dependent kinase (CDK) 4 & 6 inhibitor.
Part C only: Received prior systemic therapy for relapsed/refractory neuroblastoma.
Currently enrolled in any other clinical study involving an investigational product or non-approved use of a drug or device.
Has received an experimental treatment in a clinical trial within the last 30 days or 5 half-lives, whichever is longer. Abemaciclib, Irinotecan, Temozolomide, Dinutuximab, GM-CSF, Abemaciclib, Irinotecan, Temozolomide, Dinutuximab, GM-CSF
Relapsed Solid Tumor, Refractory Solid Tumor
Recurrent neuroblastoma, CDK4, CDK6, Ewing's sarcoma, Neuroblastoma, Malignant rhabdoid tumor, Rhabdomyosarcoma, Osteosarcoma, Brain tumor, Glioblastoma, Malignant glioma, Diffuse intrinsic pontine glioma, Medulloblastoma, Ependymoma, Solid tumor, High-grade glioma
Choline Supplementation as a Neurodevelopmental Intervention in Fetal Alcohol Spectrum Disorders Study (CHOLINE4)
This is a double-blind randomized placebo-controlled clinical trial examining choline supplementation in 2-5 year old children with Fetal Alcohol Spectrum Disorders. Outcome measures are neurocognitive tests of memory, attention, and behavior.
Jeffrey Wozniak
All
30 Months to 72 Months old
Phase 2
1506M74642
1506M74642
Inclusion Criteria:
Ages 2.5 years to 5 years old (<6 years of age) at enrollment
Prenatal alcohol exposure
Available parent or legal guardian capable of giving informed consent for participation.
Exclusion Criteria:
History of a neurological condition (ex. epilepsy, traumatic brain injury)
History of a medical condition known to affect brain function
Other neurodevelopmental disorder (ex. autism, Down syndrome)
History of very low birthweight (<1500 grams) Choline Bitartrate, Choline Bitartrate
Fetal Alcohol Spectrum Disorders
MT2022-27: TRANSPIRE: Lung Injury in a Longitudinal Cohort of Pediatric HSCT Patients
Primary Purpose: To identify risk factors and mechanisms of lung injury, test novel diagnostic strategies and treatments to reduce morbidity and mortality from lung injury after transplant.
Samuel Goldfarb
All
to 24 Years old
STUDY00016089
STUDY00016089
Inclusion Criteria:
Subjects ≤ 24 years of age undergoing allogeneic or autologous HSCT.
Exclusion Criteria:
Subjects over 24 years of age. Bronchiolitis Obliterans, Diffuse Alveolar Hemorrhage, Hematopoietic Stem Cell Transplant (HSCT), Interstitial Pneumonitis, Thrombotic Microangiopathies
A person-centered employment preparation program for adolescents and young adults with autism spectrum disorder and their families
This study includes the development and evaluation of a person-centered employment preparation program for families of transition-aged youth with autism.
Rebekah Hudock
STUDY00013507
STUDY00013507
Characterization of spleen motion and anatomy using imaging and sensors
This research is being performed to examine how the spleen moves during breathing in various body positions and breathing conditions. Physical measurements of the participant's body will be recorded (weight, height, and body dimensions) and then noninvasive recordings of the spleen and breathing patterns will be recorded. The spleen motion will be measured using standard abdominal ultrasound imaging, and breathing will be measured with accelerometers (small devices about the size of a quarter that measure the movement of the chest during breathing).
Hubert Lim
STUDY00013252
STUDY00013252
Cardiac Sarcoidosis Multi-Center Randomized Controlled Trial (CHASM CS- RCT) (CHASM-CS-RCT)
This is a Prospective, open-label, non-inferiority, randomized controlled trial (1:1 randomization) with blinded end-point analysis comparing the use of Prednisone versus Prednisone plus Methatrexate in the treatment of Cardiac Sarcoidosis.
Chetan Shenoy
All
18 Years to old
Phase III
STUDY00015181
STUDY00015181
Inclusion Criteria:
(i) Cardiac sarcoidosis presenting with one or more of the following clinical findings:
advanced conduction system disease (defined as Mobitz II AV block or third degree AV block)
significant sinus node dysfunction (defined as average HR less than 40bpm when awake and/or sustained atrial arrhythmias)
non- sustained or sustained ventricular arrhythmia
left ventricular dysfunction (LVEF < 50%)
right ventricular dysfunction (RVEF < 40%)
AND
(ii) No alternative explanation for clinical features
AND
(iii) FDG-PET uptake suggestive of active CS within two months of enrollment (confirmed by PET core lab read)
AND ONE OR BOTH OF FOLLOWING
(iv) Positive biopsy for Sarcoid (either EMB or extra-cardiac)
(v) CT Chest showing features consistent with pulmonary sarcoidosis and/or mediastinal and/or hilar lymphadenopathy
Exclusion Criteria:
Current or recent (within two months) non-topical treatment for sarcoidosis
Currently taking Methotrexate or Prednisone for another health condition
Intolerance or contra-indication to Methotrexate or Prednisone
Patient does not meet all of the above listed inclusion criteria
Patient is unable or unwilling to provide informed consent
Patient is included in another randomized clinical trial
Patient has a contraindication to PET imaging or is unlikely to tolerate due to severe claustrophobia
Pregnancy (all women of child bearing age and potential will have a negative BHCG test before enrollment)
Breastfeeding
Women of childbearing age who refuse to use a highly effective and medically acceptable form of contraception throughout the study
Patients for whom the investigator believes that the trial is not in the interest of the patient Prednisone or Prednisolone, Methotrexate, Prednisone or Prednisolone, Methotrexate
Cardiac Sarcoidosis, Sarcoidosis
Cardiac Sarcoidosis, Methotrexate, Prednisone (or Prednisolone)
EnVision CF Multicenter Study of Glucose Tolerance in Cystic Fibrosis
The purpose of this research study is because Cystic Fibrosis Related Diabetes (CFRD) has been identified by the cystic fibrosis (CF) community as one of the top ten priorities for CF research. We know that high blood sugars caused by not enough insulin lead to worse lung function in CF even before diabetes develops. However, we do not know which people with abnormal blood sugars will have long term problems.
Amir Moheet
All
6 Years to old
N/A
STUDY00004854
STUDY00004854
Inclusion Criteria:
Age >/= 6 years
Diagnosis of cystic fibrosis
CF patients regularly attending the CF centers
Clinically stable in previous 3wks:
absence of major clinical events including pulmonary exacerbations,
no change in their habitual treatment regimen including introduction of antibiotics or steroids in the past 3 weeks
Exclusion Criteria:
Diagnosis of type 1 diabetes, type 2 diabetes, or MODY
Organ transplantation
new diagnosis of CFRD in the past 6 months
antidiabetic treatment in past 6 mos (insulin or oral hypoglycemic agents)
-patients with previous CFRD diagnosis, but not currently taking insulin/glucose-lowering medications for at least 6 months should be included
pulmonary exacerbation associated with systemic steroid requirement in the last 6 months
on CFTR corrector less than 6 months prior to enrollment Oral glucose tolerance test, Continuous glucose monitoring, Dexa scan, Oral glucose tolerance test, Continuous glucose monitoring, Dexa scan
Cystic Fibrosis-related Diabetes
cystic fibrosis, insulin, glucose, children, abnormal glucose tolerance, impaired glucose tolerance, indeterminate glycemia, diabetes
Nornicotine in Smokeless Tobacco as a Precursor for Carcinogen Exposure
Smokeless tobacco users who are unable or unwilling to quit tobacco use may be exposed to the potent oral and esophageal carcinogen NNN not only from tobacco itself, but also via its endogenous synthesis from nornicotine. The proposed study will lead to an understanding of the endogenous formation of NNN from nornicotine in humans, and will also investigate the effect of the reduction of nornicotine content in smokeless tobacco on the extent of endogenous NNN formation. The knowledge gained in this study will lead to the development of recommendations for the regulation, or potentially elimination, of nornicotine in smokeless tobacco products in order to minimize exposure to NNN in the users of these products.
Irina Stepanov
All
18 Years to 65 Years old
NA
STUDY00002464
STUDY00002464
Inclusion Criteria:
Male or female adult smokeless tobacco users 18-65 years of age, using at least 3 tins of product per week for 6 months;
Used the same brand for >80% of their smokeless tobacco use over the course of at least 6 months, and used this brand exclusively for at least two weeks prior to the eligibility screening;
Not smoking or using any other nicotine or tobacco product in the past 2 weeks (expired CO < 6 ppm);
Participants are in good physical health (no unstable medical condition) and good general oral health as determined by the licensed medical professional;
Participants are in stable, good mental health (e.g. not currently, within the past 6 months, experiencing unstable or untreated psychiatric diagnosis, including substance abuse) as determined by the licensed medical professional;
Participants who are not taking any medications that affect relevant metabolic enzymes;
Women who are not pregnant or nursing or planning to become pregnant;
Participants have provided written informed consent to participate in the study.
Exclusion Criteria:
Significant immune system disorders, respiratory diseases, kidney or liver diseases or any other medical disorders that may affect biomarker data as determined by the licensed medical professional;
Vital signs out of range as determined by the licensed medical professional (participants failing for vital signs will be allowed to re-screen once):
Evident poor oral health (significant gum recession, dental caries, tooth loss) as determined by the general oral health status check;
Excessive drinking (e.g., 5 or more drinks daily) or problems with drinking or drugs (e.g., self-report of binge drinking alcohol or treatment for drug or alcohol abuse within last 3 months); to be assessed by PI or licensed medical professional;
Regular smoking or tobacco use (e.g., greater than once a week) other than oral smokeless tobacco products;
Currently (within the past 2 weeks) using nicotine replacement or other tobacco cessation products (to minimize confounding effects of another product); Herbal Snuff (Smokeless Tobacco), Herbal Snuff (Smokeless Tobacco)
Smokeless Tobacco
MT2013-34C: Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia: Standard of Care Considerations
Christen Ebens
All
0 Years to 70 Years old
N/A
1404M50183
1404M50183
Inclusion Criteria:
Aged 0 - 70 years
Acceptable hematopoeitic stem cell donor
Dyskeratosis Congenita (DC) with evidence of BM failure defined as:
requirement for red blood cell and/or platelet transfusions or
requirement for G-CSF or GM-CSF or erythropoietin or
refractory cytopenias having one of the following three
platelets <50,000/uL or transfusion dependent
absolute neutrophil count <500/uL without hematopoietic growth factor support
hemoglobin <9g/uL or transfusion dependent
Diagnosis of DC with a triad of mucocutaneous features:
oral leukoplakia
nail dystrophy
abnormal reticular skin hyperpigmentation, or
Diagnosis of DC with one of the following:
short telomeres (under a research study)
mutation in telomerase holoenzyme (DKC1, TERT, TERC, NOP10, NHP2, TCAB1)
mutation in shelterin complex (TINF2)
mutation in telomere-capping complex (CTC1)
Severe Aplastic Anemia (SAA) primary transplant with evidence of BM failure:
Refractory cytopenia defined by bone marrow cellularity <50% (with < 30% residual hematopoietic cells)
Diagnosis of SAA with refractory cytopenias having one of the following three:
platelets <20,000/uL or transfusion dependent
absolute neutrophil count <500/uL without hematopoietic growth factor support
absolute reticulocyte count <20,000/uL
Severe Aplastic Anemia (SAA) requiring a 2nd transplant
Graft failure as defined by blood/marrow chimerism of < 5%
Early myelodysplastic features
With or without clonal cytogenetic abnormalities
Adequate organ function defined as:
cardiac: left ventricular ejection fraction ≥ 35% with no evidence of decompensated heart failure
pulmonary: DLCO ≥30% predicted, no supplemental oxygen requirement
renal: Glomerular filtration rate (GFR) ≥30% predicted
Voluntary written consent
Exclusion Criteria:
Acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy
Pregnant or lactating
Uncontrolled infection
Prior radiation therapy (applies to SAA patients only)
Diagnosis of Fanconi anemia based on DEB
Diagnosis of dyskeratosis congenita with advanced MDS or acute myeloid leukemia with >30% blasts Alemtuzumab, Fludarabine, Cyclophosphamide, Total Body Irradiation, Stem Cell Transplant, Anti-thymocyte globulin, Alemtuzumab, Fludarabine, Cyclophosphamide, Total Body Irradiation, Stem Cell Transplant, Anti-thymocyte globulin
Dyskeratosis Congenita, Aplastic Anemia
Clinics and Surgery Center (CSC)
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
Male or Female
14 Years to 25 Years old
N/A
STUDY00010820
STUDY00010820
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. Anterior cruciate ligament reconstruction (ACLR), Lateral extra-articular tenodesis (LET), Anterior cruciate ligament reconstruction (ACLR), Lateral extra-articular tenodesis (LET)
Bone, Joint & Muscle
Clinics and Surgery Center (CSC)
Maternal Stress, Human Milk Composition, and Neurodevelopmental and Feeding Outcomes
This study explores the associations between maternal stress, breastmilk composition, and feeding and neurodevelopment for preterm infants in the NICU and at 4 months corrected age.
Emily Nagel
All
28 Weeks to 32 Weeks old
STUDY00016926
STUDY00016926
Inclusion Criteria:
preterm infant born between 28 0/7 and 32 6/7 weeks' gestation
mother of preterm infant meeting criteria and a) 18 to 45 years of age at the time of delivery and b) pre-gravid or first trimester BMI between 18.5 to 40 kg/m^2
Exclusion Criteria:
infants: major congenital anomalies, anticipated death, positive blood culture at birth, hypoxic ischemic encephalopathy, grade IV intraventricular hemorrhage, or plan to transfer care before discharge (35-37 weeks postmenstrual age).
mothers: a) alcohol consumption >1 drink per week or any tobacco consumption during pregnancy, b) history/current Type I or II diabetes or gestational diabetes mellitus, c) known congenital metabolic, endocrine disease or congenital illness affecting infant feeding/growth Prematurity