The main purpose of this study is to investigate efficacy, pharmacokinetics and safety of the drug in pediatric participants with moderately to severely active ulcerative colitis (UC).
The purpose of this study is to examine if it is feasible to administer decitabine and filgrastim after allogenic hematopoietic stem cell transplant (HCT) in children and young adults with
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This study will investigate the role of genetic modifiers in hemoglobinopathies through a large-scale, multi-ethnic genome-wide association study (GWAS).
The primary objective of the clinical investigation is to demonstrate successful clinical use of the ThecaFlex DRx™ System in delivering nusinersen in subjects with spinal muscular atrophy (SMA).
All
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The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo
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The proposed study includes a newborn developmental intervention to improve neurodevelopmental (ND) and medical outcomes for infants with congenital heart disease (CHD) with improved parent well-being
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The purpose of this study is to learn more about changes in glucose levels in hospitalized infants with intestinal failure receiving parenteral nutrition or PN (nutrients delivered intravenously), as
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This is a phase 2a, multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NST-6179 in subjects with
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The goal of this research study is to test the efficacy of a novel immunosuppressive agent, belumosudil, in allogeneic hematopoietic stem cell transplant (HSCT) recipients who have been newly
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The goal of this observational study is to identify candidate biomarkers in individuals with Rett Syndrome (RTT). The main questions it aims to answer are:
* Do these biomarkers change during
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Teleconsultation, or the use of video telecommunications technology to deliver expert recommendations for care remotely, has been used to improve the safety and quality of emergency care for children
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The objective of the Pediatric Gastroparesis Registry 2 is to create a national prospective registry of children, adolescents, and young adults with gastroparesis and gastroparesis-like syndrome
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The goal of this clinical trial is to determine the impact of a parental support package for new trainee mothers which focuses on mentorship, lactation, and sleep, and access to perinatal care. The
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This is a trial in which 350 primary lung transplant recipients will be randomized (1:1) to receive either Tocilizumab (six doses over 20 weeks) plus standard triple maintenance immunosuppression or
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The goal of this study is to find the best way to help participants and families manage the stress of facing a serious illness and be better able to "bounce back" or be resilient after a difficult
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COMPRENDO (ChildhOod Malignancy Peer Research NavigatiOn) is a multi-site randomized clinical trial (RCT) that uses a Hybrid Type 1 design, to test the effects of a clinical intervention on patient
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This is a pediatric kidney transplant study comparing the safety and efficacy of an immunosuppressive regimen of belatacept and sirolimus to tacrolimus and Mycophenolate Mofetil (MMF). Two hundred
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In this research study, the investigators want to learn more about the role of new innovative surgical devices, the Karl Storz Curved and Straight Fetoscopes for in-utero surgery. A fetoscope is like
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Shwachman-Diamond syndrome (SDS) is a genetic condition characterized by bone marrow failure, medical co-morbidities, and leukemia predisposition. SDS-Like patients share clinical features with SDS
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