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COG APEC1621D - NCI-COG PEDIATRIC MATCH (MOLECULAR ANALYSIS FOR THERAPY CHOICE)- PHASE 2 SUBPROTOCOL OF LY3023414 IN PATIENTS WITH SOLID TUMORS
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
NCT03213678
STUDY00001752
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
Minnesota Pulmonary Hypertension Repository Study
Pulmonary hypertension is “high pressure in the lungs.” The WHO organization classifies pulmonary hypertension into 5 subcategories: I- Pulmonary Arterial Hypertension (idiopathic or related to secondary to causes), II- Pulmonary Venous Hypertension, III- Pulmonary Hypertension Associated with the Respiratory System and or/Hypoxemia, IV- Pulmonary Hypertension due to Chronic Thrombotic and/or Embolic Disease, and V- Pulmonary Hypertension due to Disorders Directly Affecting the Pulmonary Vasculature. This registry will collect data on subjects with all types of pulmonary hypertension. The purpose of this study is to acquire information on all patients being cared for by the Pulmonary Hypertension program and entered into a repository for future research purposes or screening for new studies that become available. If a subject is eligible for an interventional study, they will be consented with an IRB approved consent form appropriate for that particular study. All other information will be used to help identify trends and hopefully lead to a better understanding of the disease progression, treatment options and outcomes.
Thenappan Thenappan
Clinical Outcomes Research
NCT00000000
1310M44485
COG ANBL1232 - Utilizing Response- and Biology-Based Risk Factors to Guide Therapy in Patients with Non-High- Risk Neuroblastoma
This phase III trial studies how well response and biology-based risk factor-guided therapy works in treating younger patients with non-high risk neuroblastoma. Sometimes a tumor may not need treatment until it progresses. In this case, observation may be sufficient. Measuring biomarkers in tumor cells may help plan when effective treatment is necessary and what the best treatment is. Response and biology-based risk factor-guided therapy may be effective in treating patients with non-high risk neuroblastoma and may help to avoid some of the risks and side effects related to standard treatment.
Emily Greengard
All
up to 18 Months old
Phase 3
NCT02176967
1410M54605
Inclusion Criteria:
• Patients must be:
• < 12 months (< 365 days) of age at diagnosis with INRG stage L1; or
• < 18 months (< 547 days) of age at diagnosis with INRG stage L2 or stage Ms neuroblastoma/ganglioneuroblastoma
• Enrollment on ANBL00B1 or APEC14B1 is required for all newly diagnosed patients
• Patients must have newly diagnosed v-myc avian myelocytomatosis viral oncogene neuroblastoma derived homolog (MYCN) non-amplified neuroblastoma (International Classification of Diseases for Oncology [ICD-O] morphology 9500/3) or MYCN non-amplified ganglioneuroblastoma verified by histology
• Patients must meet the specified criteria for one of the treatment groups defined below; genomic features include MYCN gene amplification, segmental chromosome aberrations (somatic copy number loss at 1p, 3p, 4p, or 11q or somatic copy number gain at 1q, 2p, or 17q) and deoxyribonucleic acid (DNA) index
• "Favorable" genomic features are defined by one or more whole-chromosome gains or hyperdiploid tumor (DNA index > 1) in the absence of segmental chromosome aberrations as defined above
• "Unfavorable" genomic features are defined by the presence of any segmental chromosome aberration (somatic copy number loss at 1p, 3p, 4p, or 11q or somatic copy number gain at 1q, 2p, or 17q) or diploid tumor (DNA index = 1); this includes copy neutral loss of heterozygosity (LOH)
• Only patients with MYCN non-amplified tumors are eligible for this study
• Group A: patients < 12 months (< 365 days) of age with newly diagnosed INRG stage L1 neuroblastoma/ganglioneuroblastoma who meet the following criteria:
• Greatest tumor diameter < 5 cm of adrenal or non-adrenal origin
• Patients with non-adrenal primaries are eligible, but must have positive uptake on metaiodobenzylguanidine (MIBG) scan or elevated catecholamine metabolites (urine or serum) to support the diagnosis of neuroblastoma
• No prior tumor resection or biopsy
• Group A will be further split into two subsets, which are mutually exclusive, for statistical purposes
• Group A1:
• > 6 months and < 12 months of age with an adrenal primary tumor < 5 cm in greatest diameter OR
• Patients less than 6 months of age with an adrenal primary tumor > 3.1 and < 5 cm in greatest diameter OR
• < 12 months of age with a non-adrenal primary site < 5 cm in greatest diameter
• Group A2: =< 6 months of age with an adrenal primary site and tumor =< 3.1 cm in greatest diameter.
• Group B: patients < 18 months (< 547 days) of age with newly diagnosed INRG stage L2 neuroblastoma/ganglioneuroblastoma who meet the following criteria:
• No life threatening symptoms or no impending neurologic or other organ function compromise (e.g. epidural or intraspinal tumors with existing or impending neurologic impairment, periorbital or calvarial-based lesions with existing or impending cranial nerve impairment, anatomic or mechanical compromise of critical organ function by tumor [abdominal compartment syndrome, urinary obstruction, etc.]); horner syndrome is not considered neurologic compromise
• No prior tumor resection, tumor biopsy ONLY
• Only patients with both favorable histology and favorable genomic features will remain on study as part of Group B; the institution will be notified of histologic and genomic results within 3 weeks of specimen submission on ANBL00B1 or APEC14B1
• Group C: patients < 18 months (< 547 days) of age with newly diagnosed INRG stage Ms neuroblastoma/ganglioneuroblastoma
• No prior radiotherapy or chemotherapy, with the exception of dexamethasone, which is allowed
• 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
Exclusion Criteria:
• Patients with MYCN amplified tumors are not eligible
• Group B and C patients who do not enroll on ANBL1232 within 4 weeks of definitive diagnostic procedure
• Group A and C patients, not required to undergo tumor biopsy, who do not enroll on ANBL1232 within 4 weeks of confirmatory imaging study
Drug: Carboplatin, Other: Clinical Observation, Drug: Cyclophosphamide, Drug: Doxorubicin Hydrochloride, Drug: Etoposide, Other: Laboratory Biomarker Analysis, Other: Pharmacological Study
Ganglioneuroblastoma, Localized Resectable Neuroblastoma, Localized Unresectable Neuroblastoma, Neuroblastoma
Ultrasound Treatment for Rheumatoid Arthritis Study [ULTRA Study] (ULTRA)
The research objective is to demonstrate efficacy of spleen ultrasound stimulation in the treatment of rheumatoid arthritis (RA) in a pilot study. In particular, a new wearable ultrasound device has been developed for anti-inflammatory treatment by a company called SecondWave Systems. We will measure RA disease activity, biomarkers and clinical metrics during and after an 8-week course of spleen-directed daily ultrasound treatments.
Erik Peterson
All
18 Years and over
N/A
NCT05417854
STUDY00016143
Inclusion Criteria:
• Males and females ages 18 and above
• Must carry a diagnosis of rheumatoid arthritis, as defined by the American College of Rheumatology in 2010: (https://www.rheumatology.org/Portals/0/Files/2010_revised_criteria_classification_ra.pdf)
• Classification as ?definite RA? is based on the confirmed presence of synovial thickening in at least 1 joint, absence of an alternative diagnosis that better explains the synovitis, and achievement of a total score of 6 or greater (of a possible 10) from the individual scores in 4 domains: number and site of involved joints (score range 0?5), serologic abnormality (score range 0?3), elevated acute-phase response (score range 0?1), and symptom duration (2 levels; range 0?1)
• Exhibiting symptoms or signs of inadequate inflammatory disease control according to one of two measures: a. Multidimensional HAQ score of greater than 0.3 b. DAS-28-CRP greater than 3.2 (http://www.phusewiki.org/wiki/index.php?title=Disease_Activity_Score_-_CRP_(DAS-CRP)
• Candidate participant?s rheumatoid arthritis medical therapy should be stable for two weeks leading up to the study. Moreover, participants must be willing to maintain their current medication regimen throughout the study enrollment period (in adjunct to the additional investigational ultrasound treatment)
Exclusion Criteria:
• Active bacterial or viral infection
• Pregnant women or those trying to become pregnant
• Receiving active chemotherapy or immunotherapy to treat malignancy within 30 days prior to enrollment
• Having received Rituximab monoclonal antibody medication within 30 days prior to enrollment
• Presence of an implanted device
• Asplenia
• Splenomegaly
• Ascites
• Recent abdominal surgery
• Currently participating in an investigational drug or device study
• Open wound/sores near stimulation sites
• Inability to perform minimal daily self-cares associated with feeding/dressing, according to HAQ
• Any other clinical reasons deemed by the investigators of the study in which the patient would not be an appropriate candidate for the study
Device: Splenic Ultrasound
Arthritis & Rheumatic Diseases, Immune Diseases, Rheumatoid Arthritis
Rheumatoid Arthritis
Creativity Camp Study
This study pursues the question of whether deep engagement in creative activities may benefit adolescents with depression symptoms by introducing a more flexible way of thinking, helping adolescents recognize and foster their own creative talents, and (ultimately) developing more positive views of themselves and their futures.
Kathryn Cullen
up to 18 Years old
Phase I/II
PSYCH-2021-30129
STUDY00014280
Inclusion Criteria:
age 12-17, no clinical diagnosis required (phase 1), depressive symptoms (phase 2)
Exclusion Criteria:
Any MRI contraindications, neurodevelopmental disorder, significant medical conditions that would interfere with participation, current active suicidal ideation and refusal to safety plan, history of significant neurological conditions that might confound MRI analyses. Children's Health, Mental Health & Addiction
Adolescents, Camp, Creativity, Depression, Diversity, Flexibility, Summer
Thoracentesis QI Study
All
18 Years and over
N/A
NCT05424120
Inclusion Criteria:
• Age >18
• Evidence of pleural effusion on imaging
• Clinical indication for thoracentesis
Exclusion Criteria:
• Age <18
• Standard contraindication for thoracentesis procedure
• Patients on positive pressure ventilation
• Patients who have opted out of research in EPIC
Procedure: Manual aspiration, Procedure: Wall suction, Procedure: Vacuum bottle drainage
Pleural Effusion
pleural effusion, thoracentesis, wall suction, manual aspiration, vacuum bottle drainage
Chilled Platelet Study
The purpose of this research study is to compare the transfusion of cold-stored (refrigerated) platelets to standard room temperature stored platelets. The goal of the trial is to determine whether platelets stored cold are similar or better at stopping bleeding compared to platelets stored at room temperature and, if so, to determine the maximum duration of cold storage that maintains a similar effect on bleeding
Claudia Cohn
Phase III
NCT04834414
STUDY00013172
Acute Blood Loss
bleeding, cold-stored platelets, complex cardiac surgery, hemostasis, platelets
Active Surveillance, Bleomycin, Etoposide, Carboplatin or Cisplatin in Treating Pediatric and Adult Patients With Germ Cell Tumors
This partially randomized phase III trial studies how well active surveillance, bleomycin, carboplatin, etoposide, or cisplatin work in treating pediatric and adult patients with germ cell tumors. Active surveillance may help doctors to monitor subjects with low risk germ cell tumors after their tumor is removed. Drugs used in chemotherapy, such as bleomycin, carboplatin, etoposide, and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.
Emily Greengard
All
Not specified
Phase 3
NCT03067181
STUDY00003824
Inclusion Criteria:
• There is no age limit for the low risk stratum (stage I ovarian immature teratoma and stage I non-seminoma or seminoma malignant GCT [all sites])
• Standard risk 1: Patient must be < 11 years of age at enrollment
• Standard risk 2: Patients must be >= 11 and < 25 years of age at enrollment
• Patients enrolling on one of the low risk arms must be newly diagnosed with a stage I germ cell tumor; for the standard risk arms, patients must be newly diagnosed with metastatic germ cell tumor (stage II or higher); histologic confirmation of a primary extracranial germ cell tumor in any of the categories outlined below is required of all patients at enrollment except for those who were initially diagnosed with stage I non-seminoma malignant GCT and later recur during observation post surgery off study; for these patients, if elevated tumor markers rise to > 5 x upper limit of normal (ULN) on at least 2 measurements taken at least 1 week apart, a diagnostic biopsy is not required for enrollment
• Low risk stage I immature teratoma (IT); site: ovarian; stage: Children's Oncology Group (COG) stage I, Federation of Gynecology and Obstetrics (FIGO) stage IA and IB; grade: 2 or 3; histology: pure immature teratoma (may contain microscopic foci of yolk sac tumor), mixed immature and mature teratoma, (no pathological evidence of MGCT); tumor markers: alpha-FP =< 1,000 ng/mL, beta-HCG institutional normal; all ages
• Low risk stage I non-seminoma MGCT; site: ovarian, testicular, or extragonadal; stage: COG stage I, FIGO stage IA and IB, American Joint Committee on Cancer (AJCC) testicular stage IA, IB and IS; histology: must contain at least one of the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma (pure or mixed); all ages
• Low risk stage I seminoma-MGCT; site: testicular; stage: COG stage I; AJCC testicular stage IA IB, and IS; histology: must contain at least one of the following: may contain immature/mature teratoma; may NOT contain yolk sac tumor, embryonal carcinoma, or choriocarcinoma; all ages
• Standard risk 1 (SR1); site: ovarian, testicular, or extragonadal; stage: COG stage II-IV, FIGO stage IC, FIGO stage II-IV (International Germ Cell Consensus Classification [IGCCC] criteria DO NOT apply); histology: must contain at least one of the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma; age (years) < 11
• Standard risk 2 (SR2)
• Site: ovarian; stage: COG stage II and III, FIGO stage IC, II and III; histology: must contain at least one of the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma; age (years) >= 11 and < 25
• Site: testicular; stage: COG stage II-IV, AJCC stage II, III, IGCCC good risk; histology: must contain at least one of the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma; tumor markers: must be IGCCC good risk; post op: alpha-FP < 1,000 ng/mL, beta-HCG < 5,000 IU/mL and lactate dehydrogenase (LDH) <
• 0 x normal; age (years) >= 11 and < 25
• Site: extragonadal; stage: COG stage II; histology: must contain at least one of the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma; age (years) >= 11 and < 25
• Notes:
• IGCCC criteria only apply to SR2 patients with a testicular primary tumor
• Use post-op tumor marker levels to determine IGCCC risk group
• Stage 1 seminoma patients are not eligible for the standard risk arms of the study
• For the low risk stage I non-seminoma MGCT and the standard risk arms, components of yolk sac tumor, embryonal carcinoma, or choriocarcinoma can be mixed with other forms of GCT, such as seminoma or mature or immature teratoma; if yolk sac tumor is the only malignant component present, then it must be deemed by the pathologist to be greater than a "microscopic component" of yolk sac tumor
• Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1, 2 or 3; use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age
• Organ function requirements apply ONLY to patients who will receive chemotherapy (SR1 and SR2 patients)
• Adequate renal function defined as:
• 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): (mg/dL)
• 1 month to < 6 months male: 0.4 female: 0.4
• 6 months to < 1 year male: 0.5 female: 0.5
• 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
• Total bilirubin =< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment)
• Serum glutamic-oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) or serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 2.5 x upper limit of normal (ULN) for age (for the purpose of this study, the ULN for SGPT is 45 U/L) (within 7 days prior to enrollment)
• Peripheral absolute neutrophil count (ANC) >= 1,000/mm^3 (within 7 days prior to enrollment) AND
• Platelet count >= 100,000/mm^3 (within 7 days prior to enrollment)
• Patients enrolling on the standard risk arms must be medically fit to receive protocol treatment and with no contraindications to protocol treatment
• Eligibility criteria to participate in the pilot study of the AYA-Hears instrument (patient reported outcomes [PROs] of ototoxicity) Note: participants in group 1 will not receive AGCT1531 protocol-directed therapy; all other AYA-HEARS patients must be enrolled on the AGCT1531 SR2 arm in order to participate
• >= 11 and < 25 years old at enrollment
• Able to fluently speak and read English
• Has received prior cisplatin- or carboplatin-based chemotherapy regimen for malignancy including diagnoses other than germ cell tumor
• Followed for cancer or survivorship care at one of the following institutions:
• Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
• Dana Farber/Harvard Cancer Center
• Hospital for Sick Children
• Children's Hospital of Eastern Ontario
• Oregon Health and Science University
• Seattle Children's Hospital
• Yale University
Exclusion Criteria:
• Patients with any diagnoses not listed including:
• Stage I testicular cancer patients who have undergone primary RPLND (retroperitoneal lymph node dissection)
• Pure dysgerminoma
• Pure mature teratoma
• Pure immature teratoma COG stage I, grade I
• Pure immature teratoma COG stage I, grade 2,3 with alpha-fetoprotein (AFP) >= 1000 ng/mL
• Pure immature teratoma COG stage II - IV or FIGO stage IC to IV
• "Poor risk" GCT (age >= 11 years old and COG stage IV ovarian, COG stage III or IV EG, or IGCCC intermediate or poor risk testicular), or
• Primary central nervous system (CNS) germ cell tumor
• Germ cell tumor with somatic malignant transformation
• Spermatocytic seminoma
• Patients must have had no prior systemic therapy for the current cancer diagnosis
• Patients must have had no prior radiation therapy with the exception of CNS irradiation of brain metastases; (this exception only applies to SR1 patients; any patients over age 11 with distant metastases to brain [stage IV disease] would be considered poor risk and therefore not eligible for this trial)
• Patients with significant, pre-existing co-morbid respiratory disease that contraindicate the use of bleomycin are ineligible for the standard risk arms of the trial
• Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs; a pregnancy test is required for female patients of childbearing potential; (this criteria applies ONLY to patients who will receive chemotherapy [SR1 and SR2 patients])
• Lactating females who plan to breastfeed their infants; (this criteria applies ONLY to patients who will receive chemotherapy [SR1 and SR2 patients])
• Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation; (this criteria applies ONLY to patients who will receive chemotherapy [SR1 and SR2 patients])
Other: Best Practice, Procedure: Biopsy, Procedure: Biospecimen Collection, Biological: Bleomycin Sulfate, Drug: Carboplatin, Drug: Cisplatin, Procedure: Computed Tomography, Drug: Etoposide, Procedure: Magnetic Resonance Imaging, Other: Pharmacogenomic Study, Procedure: Pulmonary Function Test, Other: Quality-of-Life Assessment, Other: Questionnaire Administration
Childhood Extracranial Germ Cell Tumor, Extragonadal Embryonal Carcinoma, Germ Cell Tumor, Malignant Germ Cell Tumor, Malignant Ovarian Teratoma, Stage I Ovarian Choriocarcinoma, Stage I Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage I Ovarian Teratoma AJCC v6 and v7, Stage I Ovarian Yolk Sac Tumor AJCC v6 and v7, Stage I Testicular Choriocarcinoma AJCC v6 and v7, Stage I Testicular Embryonal Carcinoma AJCC v6 and v7, Stage I Testicular Seminoma AJCC v6 and v7, Stage I Testicular Yolk Sac Tumor AJCC v6 and v7, Stage II Ovarian Choriocarcinoma, Stage II Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage II Ovarian Yolk Sac Tumor AJCC v6 and v7, Stage II Testicular Choriocarcinoma AJCC v6 and v7, Stage II Testicular Embryonal Carcinoma AJCC v6 and v7, Stage II Testicular Yolk Sac Tumor AJCC v6 and v7, Stage III Ovarian Choriocarcinoma, Stage III Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage III Ovarian Yolk Sac Tumor AJCC v6 and v7, Stage III Testicular Choriocarcinoma AJCC v6 and v7, Stage III Testicular Embryonal Carcinoma AJCC v6 and v7, Stage III Testicular Yolk Sac Tumor AJCC v6 and v7, Stage IV Ovarian Choriocarcinoma, Stage IV Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage IV Ovarian Yolk Sac Tumor AJCC v6 and v7, Testicular Mixed Choriocarcinoma and Embryonal Carcinoma, Testicular Mixed Choriocarcinoma and Teratoma, Testicular Mixed Choriocarcinoma and Yolk Sac Tumor
Longitudinal Study of Porphyrias
The objective is to conduct a longitudinal investigation of the natural history, complications, and therapeutic outcomes in people with acute and cutaneous porphyria.
Marshall Mazepa, MD
All
Not specified
NA
NCT01561157
STUDY00010102
Inclusion Criteria:
• Individuals with a documented diagnosis of a porphyria.
• For each type of porphyria, the inclusion criteria are based on
• Biochemical findings, as documented by laboratory reports (or copies) of porphyria-specific testing performed after 1980 (Absolute values are preferred for diagnostic biochemical thresholds. Fold increases in comparison to an upper (or lower) limit of normal (ULN or LLN) are also acceptable, but are complicated by considerable variation between laboratories in normal limits. Equivocal biochemical measurements may require confirmation by a consortium reference laboratory;)
• molecular findings documenting the identification of a mutation in a porphyria-related gene.
• In addition, an individual or a parent or guardian must be willing to give written informed consent or assent, as appropriate.
• Provision is made for enrolling relatives who may not have symptoms but have biochemical or molecular documentation of a porphyria, or in the case of recessive disorders carry a disease-related mutation.
Exclusion Criteria:
• Cases with elevations of porphyrins in urine, plasma or erythrocytes due to other diseases (i.e. secondary porphyrinuria or porphyrinemia), such as liver and bone marrow diseases;
• Patients with a prior diagnosis of porphyria that cannot be documented by review of existing medical records or repeat biochemical or DNA testing.
Acute Porphyrias, Cutaneous Porphyrias
porphyria, acute intermittent, coproporphyria, variegate, erythropoietic, protoporphyria, hepatoerythropoietic, cutanea tarda, AIP, HCP, VP, ADP, ALAD, PCT, HEP, CEP, EPP, XLP, homozygous dominant, acute hepatic, AHP
Ten Thousand Families Study
The purpose of this study is to study the evolution of early life risk factors that may lead to cancer and other conditions. This is a prospective cohort study of families who reside in Minnesota.
Logan Spector
Not specified
NA
PEDS-2017-25905
STUDY00000877
Inclusion Criteria:
• 1st Participant: 18+ living in MN
• Other family members: All ages and must live in MN, ND, SD, IA, or WI
• Participants ages 0-17 must have a parent consent to their participation and assist with study activities
Exclusion Criteria:
• Unwilling or unable to provide DNA and blood sample
• Does not have at least 1 living family member in MN IA, ND, SD, or WI
Cancer, Microbiota, Prevention & Wellness
Colon Cancer, Minnesota, PFAS, environment, exposures, family, genetics, glyphosate, lifestyle, radon, 10KFS
COG ANBL00B1 - Neuroblastoma Biology Studies
This research trial studies biomarkers in tumor tissue samples from patients with newly diagnosed neuroblastoma or ganglio-neuroblastoma. Studying samples of tumor tissue from patients with cancer in the laboratory may help doctors identify and learn more about biomarkers related to cancer.
Emily Greengard
All
up to 30 Years old
N/A
NCT00904241
0807M39682
Inclusion Criteria:
• All newly diagnosed patients with suspected neuroblastoma, suspected ganglioneuroblastoma, or suspected ganglioneuroma/maturing subtype seen at Children's Oncology Group (COG) institutions are eligible for this study
• There will be no penalty under any circumstances for enrollment of a patient whose definitive institutional diagnosis, or central review diagnosis, is found to be a tumor other than neuroblastoma, ganglioneuroblastoma, or ganglioneuroma/ maturing subtype
• Patients may not have received chemotherapy prior to enrollment on ANBL00B1 and procurement of study-related tissues with the following exception:
• Patients that in the opinion of the treating physician are too ill to undergo pre-treatment tissue biopsy and require EMERGENT chemotherapy may be enrolled on ANBL00B1; documentation of the emergent nature of therapy initiation is required
• It is required that a good faith effort (documented by specimen tracking) be made to submit a neuroblastoma sample (tumor, metastasis, and/or tumor-involved bone marrow) of sufficient quality for MYCN analysis in the Neuroblastoma Reference Laboratory in order for any newly diagnosed patient to be enrolled on ANBL00B1; this should be obtained prior to initiation of therapy
• Exceptions
• In rare cases, patients may be deemed too ill to undergo pre-treatment tissue biopsy and require EMERGENT therapy; the following eligibility guidelines apply to these cases:
• For presumed INSS stage 4S patients: Efforts to submit tumor tissue (e.g., primary tumor, skin nodule, or metastatic site) within 96 hours of EMERGENT therapy initiation should be made; however, if the child is deemed too unstable for such a procedure they may still be enrolled as long as pre-treatment peripheral blood and serum have been submitted
• For all other INSS stages: tumor tissue should be obtained as soon as possible within 96 hours of EMERGENT therapy initiation; patients without tumor tissues submitted within this time-frame are not eligible for enrollment
• Note: it may not be possible to obtain all necessary tumor biomarkers for therapy stratification in such cases; if a patient enrolled on ANBL00B1 undergoes an additional diagnostic procedure within 96 hours of initiating therapy, additional tumor specimens may be submitted to obtain biomarkers used for risk classification; the decision to perform such procedures, and/or submit these specimens, is to be made by the managing clinicians and should reflect the clinical need to know the status of such biomarkers
• Patients enrolled on ANBL1232 in Group A (either A1 or A2) will not have a tumor biopsy or resection upfront; tumor tissue submission is therefore not required for these patients to enroll on ANBL00B1; a peripheral blood and serum sample is the only specimen required to be submitted for this group of patients; should they undergo a biopsy or resection at a later date tumor can be submitted for biomarker testing at this time
• 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
Exclusion Criteria:
• Patients with relapsed neuroblastoma who were not enrolled on ANBL00B1 at original diagnosis are NOT eligible; samples should be submitted as part of the ABTR04B1 protocol
Other: Cytology Specimen Collection Procedure, Other: Laboratory Biomarker Analysis
Ganglioneuroblastoma, Localized Resectable Neuroblastoma, Localized Unresectable Neuroblastoma, Regional Neuroblastoma, Stage 4 Neuroblastoma, Stage 4S Neuroblastoma
Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening 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
NCT03155620
STUDY00001752
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
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
N/A
NCT02734277
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
Extravascular ICD Pivotal Study (EV ICD)
Henri Roukoz
All
18 Years and over
N/A
NCT04060680
STUDY00007872
Pivotal
• Patient has a Class I or IIa indication for implantation of an ICD according to the ACC/AHA/HRS Guidelines [i], or ESC guidelines [ii].
• Patient is at least 18 years of age and meets age requirements per local law.
• Patient is geographically stable and willing and able to complete the study procedures and visits for the duration of the follow-up. [i] Al-Khatib SM, Stevenson WG, Ackerman MJ, Bryant WJ, Callans DJ, Curtis AB, Deal BJ, Dickfeld T, Field ME, Fonarow GC, Gillis AM, Hlatky MA, Granger CB, Hammill SC, Joglar JA, Kay GN, Matlock DD, Myerburg RJ, Page RL. 2017 AHA/ACC/HRS guideline for management of patients with ventricular arrhythmias. [ii] Priori SG, Blomstrom-Lundqvist C, Mazzanti A, Blom N, Borggrefe M, Camm J, Elliot PM, Fitzsimons D, Hatala R, Hindricks G, Kirchhof P, Kjeldsen K, Kuck KH, Hernandez-Madrid A, Nikolaou N, Norekval TM, Spaulding C, Van Veldhuisen DJ. 2015 ESC guidelines for the management of patients with ventricular arrhythmias and the prevention of sudden cardiac death. European Heart Journal 2015 36:41 (2793-2867). https://doi.org/10.1093/eurheartj/ehv316 Pivotal
• Patient is unwilling or unable to personally provide Informed Consent.
• Patient has indications for bradycardia pacing [iii] or Cardiac Resynchronization Therapy (CRT) [iv] (Class I, IIa, or IIb indication).
• Patient with an existing pacemaker, ICD, or CRT device implant or leads.
• Patients with these medical interventions are excluded from participation in the study:
• Prior sternotomy
• Any prior medical condition or procedure that leads to adhesions in the anterior mediastinal space (i.e., prior mediastinal instrumentation, mediastinitis)
• Prior abdominal surgery in the epigastric region
• Planned sternotomy
• Prior chest radiotherapy Or any other prior/planned medical intervention not listed that precludes their participation in the opinion of the Investigator.
• Patient has previous pericarditis that:
• Was chronic and recurrent, or
• Resulted in pericardial effusion [v], or
• Resulted in pericardial thickening or calcification [vi].
• Patients with these medical conditions or anatomies are excluded from participation in the study:
• Hiatal hernia that distorts mediastinal anatomy
• Marked sternal abnormality (e.g., pectus excavatum)
• Decompensated heart failure
• COPD with oxygen dependence
• Gross hepatosplenomegaly Or any other known medical condition or anatomy type not listed that precludes their participation in the opinion of the Investigator.
• Patients with a medical condition that precludes them from undergoing defibrillation testing:
• Severe aortic stenosis
• Intracardiac LA or LV thrombus
• Severe proximal three-vessel or left main coronary artery disease without revascularization
• Hemodynamic instability
• Unstable angina
• Recent stroke or transient ischemic attack (within the last 6 months)
• Known inadequate external defibrillation
• LVEF <20%
• LVEDD >70 mm Or any other known medical condition not listed that precludes their participation in the opinion of the Investigator.
• Patient with any evidence of active infection or undergoing treatment for an infection.
• Patient is contraindicated from temporary suspension of oral/systemic anticoagulation
• Patient with current implantation of neurostimulator or any other chronically implanted device that delivers current in the body.
• Patient meets ACC/AHA/HRS or ESC clinical guideline Class III criteria for an ICD (e.g., life expectancy of less than 12 months).
• Patient is enrolled or planning to enroll in a concurrent clinical study that may confound the results of this study, without documented pre-approval from a Medtronic study manager.
• Patient with any exclusion criteria as required by local law (e.g., age or other).
• Pregnant women or breastfeeding women, or women of child bearing potential and who are not on a reliable form of birth regulation method or abstinence. [iii] 2015 HRS/EHRA/APHRS/SOLAECE expert consensus statement on optimal implantable cardioverter-defibrillator programming and testing). [iv] ACC/AHA/HRS guidelines for Cardiac Resynchronization Therapy. [v] As documented on echo or MRI. [vi] As documented on CT scan or MRI. Continued Access Inclusion Criteria
• Patient has a Class I or IIa indication for implantation of an ICD according to the ACC/AHA/HRS Guidelines.[1]
• Patient is willing and able to sign and date the Informed Consent Form for their participation in the study.
• Patient is at least 18 years of age and meets age requirements per local law.
• Patient is geographically stable and willing and able to comply with the study procedures and visits for the duration of the follow-up. [1] Al-Khatib SM, Stevenson WG, Ackerman MJ, Bryant WJ, Callans DJ, Curtis AB, Deal BJ, Dickfeld T, Field ME, Fonarow GC, Gillis AM, Granger CB, Hammill SC, Hlatky MA, Joglar JA, Kay GN, Matlock DD, Myerburg RJ, Page RL. 2017 AHA/ACC/HRS Guideline for Management of Patients With Ventricular Arrhythmias and the Prevention of Sudden Cardiac Death: A Report of the American College of Cardiology/American Heart Association Task Force on Clinical Practice Guidelines and the Heart Rhythm Society. J Am Coll Cardiol. 2018 Oct 2;72(14):e91-e220. doi: 10.1016/j.jacc.2017.10.054. Epub 2018 Aug 16. Review. Erratum in: J Am Coll Cardiol. 2018 Oct 2;72(14):1760. Continued Access Exclusion Criteria
• Patient has indications for bradycardia pacing[2] or Cardiac Resynchronization Therapy (CRT)[3] (Class I, IIa, or IIb indication).
• Patients with an existing pacemaker, ICD, or CRT device or leads.
• Patients with these medical interventions are excluded from participation in the study:
• Prior sternotomy
• Any prior medical condition or procedure that leads to adhesions in the anterior mediastinal space (i.e., prior mediastinal instrumentation, mediastinitis)
• Prior abdominal surgery in the epigastric region
• Planned sternotomy
• Prior chest radiotherapy Or any other prior/planned medical intervention not listed that precludes their participation in the opinion of the Investigator.
• Patients with these medical conditions or anatomies are excluded from participation in the study:
• Hiatal hernia that distorts mediastinal anatomy
• Marked sternal abnormality (e.g., pectus excavatum that impedes the substernal tunneling path or procedure)
• Decompensated heart failure
• COPD with oxygen dependence
• Gross hepatosplenomegaly Or any other known medical condition or anatomy type not listed that precludes their participation in the opinion of the Investigator.
• Patients with a medical condition that precludes them from undergoing defibrillation testing:
• Severe aortic stenosis
• Current Intracardiac LA or LV thrombus
• Severe proximal three-vessel or left main coronary artery disease without revascularization
• Hemodynamic instability
• Unstable angina
• Recent stroke or transient ischemic attack (within the last 6 months)
• Known inadequate external defibrillation
• LVEF < 20%
• LVEDD >70 mm Or any other known medical condition not listed that precludes their participation in the opinion of the Investigator.
• Patient with any evidence of active infection or undergoing treatment for an infection.
• Patient is contraindicated from temporary suspension of oral/systemic anticoagulation.
• Patient with current implantation of neurostimulator or any other chronically implanted device that delivers current in the body.
• Patient meets ACC/AHA/HRS or ESC clinical guideline Class III criteria for an ICD (e.g., life expectancy of less than 12 months).
• Patient is enrolled or planning to enroll in a concurrent clinical study that may confound the results of this study, without documented pre-approval from a Medtronic study manager.
• Patient with any exclusion criteria as required by local law (e.g., age or other).
• Pregnant women or breastfeeding women, or women of childbearing potential and who are not on a reliable form of birth regulation method or abstinence* (*If required by local law, women of child-bearing potential must undergo a pregnancy test within seven days prior to EV ICD Continued Access Study procedures) [2] Wilkoff BL, Fauchier L, Stiles MK, Morillo CA, Al-Khatib SM, Almendral J, Aguinaga L, Berger RD, Cuesta A, Daubert JP, Dubner S, Ellenbogen KA, Mark Estes NA 3rd, Fenelon G, Garcia FC, Gasparini M, Haines DE, Healey JS, Hurtwitz JL, Keegan R, Kolb C, Kuck KH, Marinskis G, Martinelli M, McGuire M, Molina LG, Okumura K, Proclemer A, Russo AM, Singh JP, Swerdlow CD, Teo WS, Uribe W, Viskin S, Wang CC, Zhang S. 2015 HRS/EHRA/APHRS/SOLAECE expert consensus statement on optimal implantable cardioverter-defibrillator programming and testing. Heart Rhythm. 2016 Feb;13(2):e50-86. doi: 10.1016/j.hrthm.2015.11.018. Epub 2015 Dec 1. [3] Kusumoto FM, Schoenfeld MH, Barrett C, Edgerton JR, Ellenbogen KA, Gold MR, Goldschlager NF, Hamilton RM, Joglar JA, Kim RJ, Lee R, Marine JE, McLeod CJ, Oken KR, Patton KK, Pellegrini CN, Selzman KA, Thompson A, Varosy PD. 2018 ACC/AHA/HRS Guideline on the Evaluation and Management of Patients With Bradycardia and Cardiac Conduction Delay: A Report of the American College of Cardiology/American Heart Association Task Force on Clinical Practice Guidelines and the Heart Rhythm Society. J Am Coll Cardiol. 2019 Aug 20;74(7):e51-e156. doi:
• 1016/j.jacc.2018.10.044. Epub 2018 Nov 6. Erratum in: J Am Coll Cardiol. 2019 Aug 20;74(7):1016-1018.
Inclusion Criteria:
• Patient has a Class I or IIa indication for implantation of an ICD according to the ACC/AHA/HRS Guidelines [i], or ESC guidelines [ii].
• Patient is at least 18 years of age and meets age requirements per local law.
• Patient is geographically stable and willing and able to complete the study procedures and visits for the duration of the follow-up. [i] Al-Khatib SM, Stevenson WG, Ackerman MJ, Bryant WJ, Callans DJ, Curtis AB, Deal BJ, Dickfeld T, Field ME, Fonarow GC, Gillis AM, Hlatky MA, Granger CB, Hammill SC, Joglar JA, Kay GN, Matlock DD, Myerburg RJ, Page RL. 2017 AHA/ACC/HRS guideline for management of patients with ventricular arrhythmias. [ii] Priori SG, Blomstrom-Lundqvist C, Mazzanti A, Blom N, Borggrefe M, Camm J, Elliot PM, Fitzsimons D, Hatala R, Hindricks G, Kirchhof P, Kjeldsen K, Kuck KH, Hernandez-Madrid A, Nikolaou N, Norekval TM, Spaulding C, Van Veldhuisen DJ. 2015 ESC guidelines for the management of patients with ventricular arrhythmias and the prevention of sudden cardiac death. European Heart Journal 2015 36:41 (2793-2867). https://doi.org/10.1093/eurheartj/ehv316 Pivotal
Exclusion Criteria:
• Patient is unwilling or unable to personally provide Informed Consent.
• Patient has indications for bradycardia pacing [iii] or Cardiac Resynchronization Therapy (CRT) [iv] (Class I, IIa, or IIb indication).
• Patient with an existing pacemaker, ICD, or CRT device implant or leads.
• Patients with these medical interventions are excluded from participation in the study:
• Prior sternotomy
• Any prior medical condition or procedure that leads to adhesions in the anterior mediastinal space (i.e., prior mediastinal instrumentation, mediastinitis)
• Prior abdominal surgery in the epigastric region
• Planned sternotomy
• Prior chest radiotherapy Or any other prior/planned medical intervention not listed that precludes their participation in the opinion of the Investigator.
• Patient has previous pericarditis that:
• Was chronic and recurrent, or
• Resulted in pericardial effusion [v], or
• Resulted in pericardial thickening or calcification [vi].
• Patients with these medical conditions or anatomies are excluded from participation in the study:
• Hiatal hernia that distorts mediastinal anatomy
• Marked sternal abnormality (e.g., pectus excavatum)
• Decompensated heart failure
• COPD with oxygen dependence
• Gross hepatosplenomegaly Or any other known medical condition or anatomy type not listed that precludes their participation in the opinion of the Investigator.
• Patients with a medical condition that precludes them from undergoing defibrillation testing:
• Severe aortic stenosis
• Intracardiac LA or LV thrombus
• Severe proximal three-vessel or left main coronary artery disease without revascularization
• Hemodynamic instability
• Unstable angina
• Recent stroke or transient ischemic attack (within the last 6 months)
• Known inadequate external defibrillation
• LVEF <20%
• LVEDD >70 mm Or any other known medical condition not listed that precludes their participation in the opinion of the Investigator.
• Patient with any evidence of active infection or undergoing treatment for an infection.
• Patient is contraindicated from temporary suspension of oral/systemic anticoagulation
• Patient with current implantation of neurostimulator or any other chronically implanted device that delivers current in the body.
• Patient meets ACC/AHA/HRS or ESC clinical guideline Class III criteria for an ICD (e.g., life expectancy of less than 12 months).
• Patient is enrolled or planning to enroll in a concurrent clinical study that may confound the results of this study, without documented pre-approval from a Medtronic study manager.
• Patient with any exclusion criteria as required by local law (e.g., age or other).
• Pregnant women or breastfeeding women, or women of child bearing potential and who are not on a reliable form of birth regulation method or abstinence. [iii] 2015 HRS/EHRA/APHRS/SOLAECE expert consensus statement on optimal implantable cardioverter-defibrillator programming and testing). [iv] ACC/AHA/HRS guidelines for Cardiac Resynchronization Therapy. [v] As documented on echo or MRI. [vi] As documented on CT scan or MRI. Continued Access Inclusion Criteria
• Patient has a Class I or IIa indication for implantation of an ICD according to the ACC/AHA/HRS Guidelines.[1]
• Patient is willing and able to sign and date the Informed Consent Form for their participation in the study.
• Patient is at least 18 years of age and meets age requirements per local law.
• Patient is geographically stable and willing and able to comply with the study procedures and visits for the duration of the follow-up. [1] Al-Khatib SM, Stevenson WG, Ackerman MJ, Bryant WJ, Callans DJ, Curtis AB, Deal BJ, Dickfeld T, Field ME, Fonarow GC, Gillis AM, Granger CB, Hammill SC, Hlatky MA, Joglar JA, Kay GN, Matlock DD, Myerburg RJ, Page RL. 2017 AHA/ACC/HRS Guideline for Management of Patients With Ventricular Arrhythmias and the Prevention of Sudden Cardiac Death: A Report of the American College of Cardiology/American Heart Association Task Force on Clinical Practice Guidelines and the Heart Rhythm Society. J Am Coll Cardiol. 2018 Oct 2;72(14):e91-e220. doi: 10.1016/j.jacc.2017.10.054. Epub 2018 Aug 16. Review. Erratum in: J Am Coll Cardiol. 2018 Oct 2;72(14):1760. Continued Access Exclusion Criteria
• Patient has indications for bradycardia pacing[2] or Cardiac Resynchronization Therapy (CRT)[3] (Class I, IIa, or IIb indication).
• Patients with an existing pacemaker, ICD, or CRT device or leads.
• Patients with these medical interventions are excluded from participation in the study:
• Prior sternotomy
• Any prior medical condition or procedure that leads to adhesions in the anterior mediastinal space (i.e., prior mediastinal instrumentation, mediastinitis)
• Prior abdominal surgery in the epigastric region
• Planned sternotomy
• Prior chest radiotherapy Or any other prior/planned medical intervention not listed that precludes their participation in the opinion of the Investigator.
• Patients with these medical conditions or anatomies are excluded from participation in the study:
• Hiatal hernia that distorts mediastinal anatomy
• Marked sternal abnormality (e.g., pectus excavatum that impedes the substernal tunneling path or procedure)
• Decompensated heart failure
• COPD with oxygen dependence
• Gross hepatosplenomegaly Or any other known medical condition or anatomy type not listed that precludes their participation in the opinion of the Investigator.
• Patients with a medical condition that precludes them from undergoing defibrillation testing:
• Severe aortic stenosis
• Current Intracardiac LA or LV thrombus
• Severe proximal three-vessel or left main coronary artery disease without revascularization
• Hemodynamic instability
• Unstable angina
• Recent stroke or transient ischemic attack (within the last 6 months)
• Known inadequate external defibrillation
• LVEF < 20%
• LVEDD >70 mm Or any other known medical condition not listed that precludes their participation in the opinion of the Investigator.
• Patient with any evidence of active infection or undergoing treatment for an infection.
• Patient is contraindicated from temporary suspension of oral/systemic anticoagulation.
• Patient with current implantation of neurostimulator or any other chronically implanted device that delivers current in the body.
• Patient meets ACC/AHA/HRS or ESC clinical guideline Class III criteria for an ICD (e.g., life expectancy of less than 12 months).
• Patient is enrolled or planning to enroll in a concurrent clinical study that may confound the results of this study, without documented pre-approval from a Medtronic study manager.
• Patient with any exclusion criteria as required by local law (e.g., age or other).
• Pregnant women or breastfeeding women, or women of childbearing potential and who are not on a reliable form of birth regulation method or abstinence* (*If required by local law, women of child-bearing potential must undergo a pregnancy test within seven days prior to EV ICD Continued Access Study procedures) [2] Wilkoff BL, Fauchier L, Stiles MK, Morillo CA, Al-Khatib SM, Almendral J, Aguinaga L, Berger RD, Cuesta A, Daubert JP, Dubner S, Ellenbogen KA, Mark Estes NA 3rd, Fenelon G, Garcia FC, Gasparini M, Haines DE, Healey JS, Hurtwitz JL, Keegan R, Kolb C, Kuck KH, Marinskis G, Martinelli M, McGuire M, Molina LG, Okumura K, Proclemer A, Russo AM, Singh JP, Swerdlow CD, Teo WS, Uribe W, Viskin S, Wang CC, Zhang S. 2015 HRS/EHRA/APHRS/SOLAECE expert consensus statement on optimal implantable cardioverter-defibrillator programming and testing. Heart Rhythm. 2016 Feb;13(2):e50-86. doi: 10.1016/j.hrthm.2015.11.018. Epub 2015 Dec 1. [3] Kusumoto FM, Schoenfeld MH, Barrett C, Edgerton JR, Ellenbogen KA, Gold MR, Goldschlager NF, Hamilton RM, Joglar JA, Kim RJ, Lee R, Marine JE, McLeod CJ, Oken KR, Patton KK, Pellegrini CN, Selzman KA, Thompson A, Varosy PD. 2018 ACC/AHA/HRS Guideline on the Evaluation and Management of Patients With Bradycardia and Cardiac Conduction Delay: A Report of the American College of Cardiology/American Heart Association Task Force on Clinical Practice Guidelines and the Heart Rhythm Society. J Am Coll Cardiol. 2019 Aug 20;74(7):e51-e156. doi:
• 1016/j.jacc.2018.10.044. Epub 2018 Nov 6. Erratum in: J Am Coll Cardiol. 2019 Aug 20;74(7):1016-1018.
Device: Defibrillation using the Extravascular ICD
Tachycardia, Ventricular Arrythmia
Clinics and Surgery Center (CSC)
A Natural History Study of the Gangliosidoses
This study's primary aims are to define and characterize disease progression for the infantile and juvenile forms of the gangliosidoses, and the late-onset forms of gangliosidosis, including their heterogeneity; and to observe treatment outcomes for any treatments tried. The secondary aims of this study are to understand the neurological involvement in late-onset gangliosidosis; and to collect data on disease progression that can be used for creation of an objective disease stage and severity index.
Jeanine Jarnes
All
Not specified
N/A
NCT00668187
1007M85712
Inclusion Criteria:
• Subjects must have a documented gangliosidosis disease.
• Subjects must be able to complete appropriate neuropsychological and neurobehavioral assessments.
• Late-onset gangliosidosis subjects must be able to tolerate a head MRI.
Exclusion Criteria:
• There are no exclusion criteria, beyond a desire not to participate.
Tay-Sachs Disease, Sandhoff Disease, Late Onset Tay-Sachs Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
Tay-Sachs disease, Sandhoff disease, Late Onset Tay-Sachs disease, LOTS, hexosaminidase A deficiency, hexosaminidase A and B deficiency, infantile Tay-Sachs disease, adult-onset Tay-Sachs disease, prospective, natural history, GM1 gangliosidosis, gangliosidoses, β-galactosidase, β-galactosidase deficiency, hexosaminidase, hexosaminidase deficiency, Tay-Sachs, Sandhoff, juvenile Tay-Sachs, juvenile Tay-Sachs disease, late onset Tay-Sachs, juvenile Sandhoff, juvenile Sandhoff disease, GM2 gangliosidosis
Microbiome and Immunosuppression: The Mission Study (MISSION)
All
18 Years and over
NCT04953715
Inclusion Criteria:
• 1) Participants undergoing kidney transplant 2) Male or female at least 18 years of age at time of enrollment 3) Will or have received a living or deceased donor kidney transplant 4) Planned post-transplant immunosuppression regimen of mycophenolate mofetil dosed every 12 hours (Cellcept or generic) and immediate release tacrolimus dosed twice daily with trough concentration monitoring (generic or brand formulation). 5) Able and willing to complete study-related procedures and visits 6) Signs written informed consent
Exclusion Criteria:
• 1) Recipient of a previous non-kidney transplant 2) Subject is a multi-organ transplant recipient 3) Presence of active gastroparesis and documented in medical record 4) Liver dysfunction (total bilirubin >2x upper limit of normal) within 2 months of enrollment 5) Patients that take medications that significantly inhibit UGT enzymes. 6) Patients that take medications that significantly inhibit or induce the biliary transporters 7) Patient is known to be HIV positive 8) Pregnant or nursing (lactating) women 9) Non-English speaking 10) Patients who have undergone bariatric surgery
Other: collection of body microbioma
Kidney Transplant Rejection, Kidney Transplant
transplant, kidney, Microbiome
Natural History Study for DNA Repair Disorders
A single-center, single-arm, non-interventional natural history study to evaluate the longitudinal clinical course, functional outcome measures, and candidate biomarkers for individuals with DNA repair disorders, including Cockayne syndrome (CS), xeroderma pigmentosum (XP), and trichothiodystrophy (TTD).
Peter Kang
All
6 Months and over
Phase I
NCT05484570
STUDY00015911
Inclusion Criteria:
• Diagnosis of Cockayne syndrome (CS), xeroderma pigmentosum (XP), or trichothiodystrophy (TTD), based on genetic testing and/or key clinical characteristics l characteristics
• Has one or more of the following neurodevelopmental or neurological complications
• Gross motor delay (non-ambulatory or started walking after age 18 months)
• Language delay (non-verbal or started talking after 18 months)
• Altered muscle tone (hypertonia, dystonia, hypotonia)
• Gait difficulties, including stiff gait, short stride, frequent falls, use of orthotics, use of walker
• Tremors
• Microcephaly
• Is a family member of an individual with the above condition
• No restrictions regarding current ambulatory status
• Minimum age for enrollment eligibility will be 6 months due to fragility of neonates with severe forms of DNA repair disorders and limitations of motor assessment scales in infants younger than 6 months. There will be no maximum age for enrollment eligibility.
• No restrictions regarding gender, race, or ethnicity.
• Voluntary written consent from the participant if adult capable of consenting or parent/guardian if minor or not capable of consenting
• Written consent of Legally Authorized Representative if enrolling adult lacks capacity to consent
Exclusion Criteria:
• Any prior history of systemic gene or cell-based therapy
• Current participation in an interventional clinical trial
Other: Interval History, Other: Physical Examination, Other: ECAB Assessment, Other: Gait Assessment, Other: Specimen Sample Collection
DNA Repair Disorder, Cockayne Syndrome, Xeroderma Pigmentosum, Trichothiodystrophy
Stratifying Patient Immune Endotypes in Sepsis (SPIES Study)
This is a continuation of an ongoing study (also called SPIES Study) which has previously been approved by the UMN IRB. We are now transferring to a central IRB at UFL following funding from the NIH. Description below: Prolonged hospitalization, secondary infections, sepsis recidivism and hospital readmission are the most common long-term consequences in sepsis survivors, but we have only limited means to diagnose immunosuppressed endotype. Here we propose to validate an ELISpot assay as an optimal biomarker to early diagnose immunosuppression, and to ex vivo test potential immune stimulants for subsequent clinical development.
Thomas Griffith
18 Years and over
NA
URO-2021-29535
STUDY00017412
Inclusion Criteria:
• All adults (age ?18)
• Ability to obtain Informed Consent prior to blood collection.
Exclusion Criteria:
• Current, chronic steroid use
• Pregnancy
• Current or recent (within 7 days) use of antibiotics.
Immune Diseases
Sepsis
The Lysosomal Disease Network Longitudinal Study of the Mucopolysaccharidoses
The purpose of this study is to examine the changes in the central nervous system over time in both treated and untreated patients with MPS I, II, IV, VI, and VII in both structure and function (including emotional-social characteristics) through the use of brain MRI, neuropsychological testing, and gathering an updated medical history.
Chester Whitley, MD, PhD
All
6 Years and over
N/A
NCT01870375
STUDY00014153
Inclusion Criteria:
• Any MPS I, II, IV, VI or VII child or adult aged 6 years of age or older
Exclusion Criteria:
• Exclusion Criteria for Neuroimaging:
• Participants with:
• Pacemakers
• Any indwelling electronic device including programmable shunts
• Orthodontic braces unless they are not made of metal
• Other implanted metal in the body other than titanium
• Unable to stay still during MRI because of low cognitive function, behavioral dysregulation, or young age, if the patient is not a clinical patient having sedation/anesthesia
• Pregnancy
• Exclusion Criteria for Neuropsychological and Neurobehavioral Testing
• Participants who:
• Are too functionally impaired for testing
Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type VI, Mucopolysaccharidosis Type IV, Mucopolysaccharidosis Type VII
Mucopolysaccharidosis, Longitudinal, Brain, Cognition, Quality-of-Life, Hurler syndrome, Hunter syndrome, Hurler-Scheie syndrome, Scheie syndrome, Maroteaux-Lamy syndrome, MPS I, MPS II, MPS VI, Mucopolysaccharidosis type I, Mucopolysaccharidosis type II, Mucopolysaccharidosis type VI, MPS IV, MPS VII, Mucopolysaccharidosis type IV, Mucopolysaccharidosis type VII, Morquio syndrome, Sly syndrome
MultiStem Administration for Stroke Treatment and Enhanced Recovery Study
This study is evaluating the efficacy of MultiStem (drug) on functional outcome in participants with ischemic stroke.
Muhammad Affan
Phase III
NCT03545607
STUDY00008000
Ischemic Stroke
adult stem cells, ischemic stroke
SynerFuse Spinal Fusion and Neuromodulation Proof of Concept Study
This study will test the completion of a fusion and implantation of a neuromodulation device during the same open back procedure.
Michael Park
18 Years and over
Pilot
NEUROSURG-2018-27335
STUDY00004880
Inclusion Criteria:
Description: This is a 12-month long study of patients between ages 21-79 years old with chronic lower back pain who have been told by a doctor that they should have a single level spinal fusion surgery to address their pain. Despite successful surgery, up to 40% of patients who have had a spinal fusion surgery can experience long-term residual back pain. This study is trying to figure out if this pain can be addressed in a safe and effective way earlier by combining two established treatments into a single procedure. It involves implanting a neuromodulation device during the spinal fusion procedure and starting the neurostimulation during the hospital stay, instead of implanting the device during a separate procedure that typically happens years later. Chronic Lower Back Pain, DRG, Neurostimulation, Spinal Fusion
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 and over
N/A
NCT03650712
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
Diagnostic Test: Oral glucose tolerance test, Diagnostic Test: Continuous glucose monitoring, Diagnostic Test: Dexa scan
Cystic Fibrosis-related Diabetes
cystic fibrosis, insulin, glucose, children, abnormal glucose tolerance, impaired glucose tolerance, indeterminate glycemia, diabetes
Prospective Evaluation of Chronic Pancreatitis for Epidemiologic and Translational Studies
This study will provide the most accurate and reliable estimates to date on disease progression and clinical events in evolving chronic pancreatitis. We also hope to develop from the results of this study some lab tests that will help us with early diagnosis of chronic pancreatitis and also to discover any genetic factors that may affect your chances of developing chronic pancreatitis.
Melena Bellin
NA
NCT03099850
STUDY00012537
Pancreatitis
Chronic Pancreatitis, Data management, Protocol monitoring, Regulatory compliance
TRE in Type 2 Diabetes (See Food Study 3) (SFS3)
Feasibility study to test our overall hypothesis that time restricted eating (TRE) presents a viable alternative to caloric restriction for improving glycemic measures and reducing weight in overweight/obese patients with metformin-only treated Type 2 diabetes (T2DM).
Lisa Chow
All
18 Years to 50 Years old
N/A
NCT05290246
STUDY00014853
Inclusion Criteria:
• Overweight/obese adults with metformin-only treated type 2 diabetes
• 18-50 years old
• BMI:25-35 kg/m2
• HbA1c: 6.5-8.5%
• duration of diabetes ≤10 years
• Self-reported weight must be stable [±5 pounds] for at least 3 months prior to the study
• Owns a smartphone.
Exclusion Criteria:
• Active or anticipated pregnancy during the study
• type 2 diabetes treated with medications other than metformin
• presence of eating disorders as noted by screening survey.
Behavioral: Time restricted eating, Behavioral: Caloric reduction
Type2Diabetes
Type 2 diabetes, diet intervention, intermittent fasting, caloric restriction, time restricted eating
Study Clinical diagnosis and functional implications of shoulder multidirectional instability
The purpose of the study is to detect subgroups of shoulder movement abnormalities in individuals with shoulder pain. The study is a comparative, cross sectional design. A clinical exam will be used to identify subgroups of subjects with specific movement abnormalities. These individuals' joint movements will be recorded utilizing dynamic fluoroscopy (x-ray) images to determine if there are distinct verifiable movement distinctions
Paula Ludewig
18 Years and over
NA
PMR-2021-30184
1603M85761
Inclusion Criteria:
Ages 18-60 with shoulder pain
Exclusion Criteria:
History of shoulder surgery or traumatic injury Bone, Joint & Muscle
motion analysis, pain, physical therapy, rehabilitation, shoulder
Advancing Treatment for Pancreatitis: A Prospective Observational Study of TPIAT (POST)
Aim 1: To determine (1a) whether patient and disease characteristics are associated with favorable pain and health-related quality-of-life outcomes (HRQOL) after TPIAT; (1b) the optimal timing of the TPIAT intervention to resolve pain and improve HRQOL; and (1c) in a subset of patients, the impact of central sensitization on pain resolution. Aim 2: To determine (2a) whether patient and disease characteristics are associated with favorable glycemic outcomes from the IAT procedure; and (2b) the optimal timing of TPIAT to obtain post-surgical insulin independence. Aim 3: To determine the cost-effectiveness of TPIAT.
Melena Bellin
All
Not specified
N/A
NCT03260387
1512M81750
Inclusion Criteria:
• Any patient with chronic or recurrent acute pancreatitis undergoing total or completion pancreatectomy with islet autotransplantation at a participating center.
Exclusion Criteria:
• Partial pancreatectomy
• TPIAT performed for a diagnosis other than chronic or recurrent acute pancreatitis (for example benign or malignant pancreatic tumor)
Procedure: TPIAT
Pancreatitis, Chronic, Pancreatectomy, Hyperglycemia, Pancreatitis
pediatric, total pancreatectomy, acute pancreatitis, chronic pancreatitis, islet transplant, islet autotransplant, Clinics and Surgery Center (CSC)
Precision Anesthesia: A Genomics Study of Post Cesarean Section Narcotic Requirements
This is planned as a multicenter project that will enroll 25 elective Cesarean section patients and gather demographic data, perioperative opioid requirements and perform genetic testing to seek preliminary evidence linking the extremes of opioid requirements (high and low) to genetics. This is intended as a pilot project to demonstrate proof of concept and the feasibility of a much larger multicenter study, with the eventual goal of defining the genomics of postoperative opioid requirements needed for pain control.
Ferne Braveman
18 Years and over
NA
ANES-2020-28514
STUDY00008680
Inclusion Criteria:
• 1
Inclusion Criteria:
Pregnant patients (ages 18 to 40) undergoing elective c-section delivery
• 2
Exclusion Criteria:
Inability to consent
Emergency C-sections (e.g. for fetal distress, placental abruptions, or failure to progress).
C-sections requiring a planned general anesthetic.
C-sections requiring non-standard surgical incisions (vertical as opposed to horizontal incisions which would be expected to alter the postoperative pain profile) Women's Health
C-Section
RPC01-3202: INDUCTION STUDY #2 - A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY OF ORAL OZANIMOD AS INDUCTION THERAPY FOR MODERATELY TO SEVERELY ACTIVE CROHN S DISEASE
This is a Phase 3, randomized, double-blind, placebo-controlled study to determine the effect of oral ozanimod as an induction treatment for subjects with moderately to severely active CD, defined as a CDAI score ≥ 220 to ≤ 450. Approximately 600 subjects with active clinical symptoms and mucosal inflammation will be randomized in a 2:1 ratio to receive either ozanimod or placebo. Subjects will be stratified by prior biologic use and corticosteroid use at baseline. Approximately 50% of subjects with a history of treatment with marketed biologic agents (eg, TNF antagonists, anti-IL-12/23 and anti-integrin therapy) will be recruited. This limit will ensure the enrollment of subjects who have failed or been intolerant to corticosteroids or immunomodulators but never failed a TNF antagonist.
Eugenia Shmidt
All
18 Years to 75 Years old
Phase 3
NCT03440385
STUDY00003227
For more information regarding Bristol-Myers Squibb Clinical Trial participation, please
visit: www.BMSStudyConnect.com
• Crohn's disease for ≥ 3 months on endoscopy and on histological exam
• Inadequate response or loss of response to corticosteroids, immunomodulators, and/or biologic therapy
• Crohn's Disease Activity Index (CDAI) score ≥ 220 and ≤ 450
• Average daily stool frequency ≥ 4 points and/or an abdominal pain of ≥ 2 points
• Simple Endoscopic Score for Crohn's Disease (SES-CD) score of ≥ 6 (or SES-CD ≥ 4 in participants with isolated ileal disease)
• Diagnosis of ulcerative colitis, indeterminate colitis, radiation colitis, or ischemic colitis, or has strictures with prestenotic dilatation requiring procedural intervention
• Extensive small bowel resection (>100cm) or known diagnosis of short bowel syndrome, or requires total parenteral nutrition
• Current stoma, ileal-anal pouch anastomosis, or fistula Other protocol-defined inclusion/exclusion criteria apply
Inclusion Criteria:
• Crohn's disease for ≥ 3 months on endoscopy and on histological exam
• Inadequate response or loss of response to corticosteroids, immunomodulators, and/or biologic therapy
• Crohn's Disease Activity Index (CDAI) score ≥ 220 and ≤ 450
• Average daily stool frequency ≥ 4 points and/or an abdominal pain of ≥ 2 points
• Simple Endoscopic Score for Crohn's Disease (SES-CD) score of ≥ 6 (or SES-CD ≥ 4 in participants with isolated ileal disease)
Exclusion Criteria:
• Diagnosis of ulcerative colitis, indeterminate colitis, radiation colitis, or ischemic colitis, or has strictures with prestenotic dilatation requiring procedural intervention
• Extensive small bowel resection (>100cm) or known diagnosis of short bowel syndrome, or requires total parenteral nutrition
• Current stoma, ileal-anal pouch anastomosis, or fistula Other protocol-defined inclusion/exclusion criteria apply
Drug: Ozanimod, Other: Placebo
Crohn Disease, Digestive & Liver Health
Crohn's Disease, Crohn Disease, Oral, Ozanimod, Moderately active, Severely active, RPC01, RPC01-3202, Clinics and Surgery Center (CSC), inflammatory bowel disease
The TrialNet Natural History Study of the Development of Type 1 Diabetes
Antoinette Moran
All
30 Months to 45 Years old
N/A
NCT00097292
0305M47349
Inclusion Criteria:
• Individuals 2.5 to 45 years old who have an immediate family member with type 1 diabetes (such as a child, parent, or sibling)
• Individuals 2.5-20 years old who have an extended family member with type 1 diabetes (such as a cousin, niece, nephew, aunt, uncle, grandparent, or half-sibling)
• Individuals 2.5-45 years old without a type 1 diabetes proband, who are known to have 1 or more islet antibody are eligible for screening if needed to determine eligibility for a clinical trial to delay or prevent disease progression.
Exclusion Criteria:
To be eligible a person must not:
• Have diabetes already
• Have a previous history of being treated with insulin or oral diabetes medications.
• Currently be using systemic immunosuppressive agents (topical and inhaled agents are acceptable)
• Have any known serious diseases
Diabetes Mellitus, Type 1
"at risk" for developing type 1 diabetes, T1DM, T1D, juvenile diabetes, Type 1 Diabetes TrialNet, TrialNet
Adaptive Phase II Study to Evaluate the Safety & Efficacy of NaBen®
All
12 Years to 17 Years old
Phase 2/Phase 3
NCT01908192
Inclusion Criteria:
• Male or female subjects who are between 12 and 17 years of age inclusive
• Physician confirmed DSM-IV or -V diagnosis of schizophrenia based on MINI International Neuropsychiatric Interview for Schizophrenia and Psychotic Disorders Studies for Children and Adolescents, version 6.0 (MINI-KID, Version 6.0)
• Are clinically stable with residual symptoms, defined as a total score of ≥ 60 of PANSS and a score of ≥ 40 for SANS
• An unchanged antipsychotic medication regimen for at least eight (8) weeks prior to randomization into the study and expected to remain unchanged during the study (longer for depot or long-acting antipsychotics: ten (10) months for Aripiprazole (Maintena®) and Paliperidone (Xeplion®); six (6) months for Olanzapine pamoate monohydrate (Zypadhera®); and at least 6 times duration of the reported half life or minimum four (4) months for other depot or long-acting antipsychotics)
• In good general physical health and all physical exam, neurological exam and laboratory assessments (urine/blood routine, biochemical tests and ECG) are clinically unremarkable per the investigator
• Subject has a negative urine illicit drug screening test
• Subject understands and is willing to sign the Informed Assent Form (IAF) prior to study entry and agrees to be available for all the study visits
• The subject's guardian understands and is willing to sign the Informed Consent Form (ICF) prior to study entry and agrees to be available for all the study visits
• Must not be a danger to self or others and must have family support available to be maintained as outpatients
Exclusion Criteria:
• Meets the DSM-IV or -V criteria at screening for mental retardation, dissociative disorder, bipolar disorder, major depressive disorder, schizoaffective disorder, schizophreniform disorder, autistic disorder, or primary substance induced psychotic disorder. Other comorbid disorders; e.g., attention-deficit hyperactivity disorder (ADHD), are allowed as long as schizophrenia is the primary diagnosis and the comorbid disorder(s) do not require medication.
• Subjects whose illness was resistant to antipsychotics according to prior trials of two different antipsychotics of adequate dose
• History of epilepsy, head trauma, or neurological illness other than Tourette's syndrome
• History of allergic reaction to sodium benzoate
• Serious medical illnesses such as acute or chronic renal disease, liver failure or heart disease that, in the opinion of the investigator, may interfere with the conduct of the study.
• Current substance abuse or positive urine illicit drug screening or history of substance dependence (including alcohol, but excluding nicotine and caffeine) in the past three (3) months.
• Use of depot antipsychotics in the past six (6) months
• Inability to follow protocol
• Body Mass Index (BMI) > 35
• Female subjects who are pregnant (as confirmed by urine pregnancy test performed at screening Visit) or are nursing, or who do not agree to abstinence or birth control during the study
• Cancer within the last three (3) years except for basal cell carcinoma and squamous cell carcinoma
• Previous participation in an intervention trial within 30 days of randomization
• Subjects whose PANSS score has decreased more than 10 percent during the Screening Phase
Drug: NaBen®, Drug: Placebo
Schizophrenia
Sodium Benzoate, Schizophrenia, Adolescent, Antipsychotic, Anti-psychotic, NMDA, NaBen, pediatric