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Collaborative Registry to Determine the Natural History of Barth Syndrome
The purpose of this research study is to collect data that we hope will help answer those questions and improve outcomes for children with Barth ...Phase N/A
Non-Interventional
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FMT and Other Gut Microbial Therapies National Registry
A national data registry of patients receiving fecal microbiota transplantation (FMT) or other gut-related-microbiota products designed to prospectively assess short and long-term safety and effectiveness.Phase N/A Non-Interventional
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A single-center observational study of whole and regional brain volumes in patients with pediatric-onset demyelinating diseases
The purpose of this research study is to better understand how the brains of children and young adults with demyelinating diseases, such as multiple sclerosis ...Phase N/A Non-Interventional
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Prospective Registry-Based Study Evaluating the Effectiveness and Safety of Odevixibat in Participants with Alagille Syndrome (ALGS)
The purpose of this research study is to gather information on how well Bylvay (Odevixibat) works for treating Alagille syndrome using real-life data collected over ...Phase N/A Non-Interventional
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Children s Awareness and Long-Term Quality of Life: A Mixed Methods Study of Pediatric Critical Care Unit (PICU) Experiences
The purpose of this study is to learn more about memories and/or experiences that children may have had during life threatening illness in the ...Phase N/A Non-Interventional
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Site for PNOC027; A pilot trial of real time drug screening and genomic testing to determine an individualized treatment plan in children and young adults with relapsed medulloblastoma
Site for The current study will use a new treatment approach based on the molecular characteristics of each participant's tumor. The study will test ...Phase N/A
Interventional
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A Phase 3 Randomized Double-Blind Dose-Ranging Evaluation of Transcutaneous Vagus Nerve Stimulation (tVNS) to Reduce Temper Outbursts in People with Prader-Willi Syndrome (PWS)
The purpose of this trial are to determine the acceptability, safety, and effectiveness of Transcutaneous Vagus Nerve Stimulation (tVNS) to treat the severe behavior problems ...Phase N/A Interventional
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Characteristics and Outcome of Fontan Associated Liver Disease in Children
The purpose of this research study is to learn more about Fontan-associated liver disease (FALD) in patients who have had Fontan heart surgery.Phase N/A Non-Interventional
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AALL2131: An International Pilot Study of Chemotherapy and Tyrosine Kinase Inhibitors with Blinatumomab in Patients with Newly-Diagnosed Philadelphia Chromosome-Positive or ABL-class Philadelphia Chromosome-Like B-cell Acute Lymphoblastic Leukemia
This pilot trial assesses the effect of the combination of blinatumomab with dasatinib or imatinib and standard chemotherapy for treating patients with Philadelphia chromosome positive ...Phase N/A Interventional
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Exploring the Association Between the Caregiver-Child Relationship and Cardiovascular Health
This is an observational study that seeks to collect data (survey, medical record, semi-structured interview, and biospecimen data) from adults who are caregivers of young ...Phase N/A Non-Interventional
Accepts Healthy Volunteer
