The purpose of this study is to determine the safety and maximum effective dose (MED) of Interleukin-2 in subjects with moderate-to-severe crohn's disease.
This is a multicenter randomized, double blind, Phase 2 trial for patients receiving transplants from 7 of 8 HLA matched donors, in which an extended dosing regimen of abatacept, and a short-term
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This is a prospective, single-arm non-randomized interventional study of fetuses to assess the safety and efficacy of fetal embolization of Vein of Galen Malformation (VOGM). Subjects will receive a
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Our goal is to conduct a large multi-site randomized controlled trial (RCT) of a promising computer-facilitated Screening and clinician Brief Intervention (cSBI) system designed for delivery by
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This observational study will examine the effects of thickened feeds on clinical outcomes and healthcare utilization in infants with brief resolved unexplained event (BRUE).
This study will examine the effects of varying liquid viscosity on swallow physiology in infants with oropharyngeal dysphagia and brief resolved unexplained event (BRUE) and other children with
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The goal of this study is to conduct a prospective, longitudinal natural history study of children and adults with Angelman Syndrome using investigator-observed and parent-reported outcome measures to
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This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators and their impact on children
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The purpose of this research study is to learn more about variants in the TP53 gene both associated with Li-Fraumeni Syndrome (LFS), a hereditary cancer risk condition, and TP53 variants found in the
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This is a phase II, prospective, longitudinal, multi-center trial of poly-ICLC (Hiltonol ®) treatment for progressive low-grade gliomas in pediatric patients with NF1. The primary objective is to
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This is a prospective observational study designed to evaluate Immature Platelet Fraction or Immature Platelet Count and Platelet Function Analyzer-100/200 Closure Time-ADP (in vitro bleeding time) as
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The incidence of moderate to severe congenital heart disease (CHD) in the United States is estimated to be 6 per 1000 live-born full term infants. Recent advances in pediatric cardiology, surgery and
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This study proposes to quantify and describe the quality of life of children with intestinal failure, and to identify the medical and socio-economic factors that impact this quality of life, using
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Open-label, dose escalation, multi-center, Phase I/II clinical trial to assess the safety/tolerability and determine the recommended Phase II Dose (RP2D) of ET140203 T-cells in pediatric subjects who
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Background:
During the first few decades of life, the brain changes dramatically in shape and function. Sleep lets researchers measure these changes. Researchers want to create a database of sleep
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This study primarily aims to determine whether patients diagnosed with osteochondritis dissecans (OCD) of the knee experience psychological stress due to their diagnosis and treatment plan.
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The FARE Patient Registry will serve as a prospective, observational food allergy reporting system that stores detailed health and other basic information about patients' real-world experiences with
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AZ-SWED is a parallel group, double blind, placebo control efficacy clinical trial with two separate hypotheses. The trial will compare the 5-day outcome of preschool children presenting to an
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The Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia (HSP) is focused on gathering longitudinal clinical data as well as biological samples (skin and/or blood and/or
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