447 | Term | Boston Children's Hospital

Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients

Red Blood Cell - IMProving trAnsfusions for Chronically Transfused recipients (RBC-IMPACT) is an observational cohort study to assess donor, component, and recipient factors that contribute to RBC ...

Intervening in Food Insecurity to Reduce and Mitigate (InFoRM) Childhood Obesity

The goals of this study are to 1) pilot the feasibility of a novel meal kit delivery intervention in families and children with food insecurity and obesity and 2) evaluate the implementation of the ...

Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy

This is a Phase 3, global, double-blind, randomized, placebo-controlled study of adjunctive GNX treatment in children and adults with TSC-related epilepsy. The study consists of a 4-week prospective ...

A Phase 3 Study to Assess the Safety and Efficacy of Pitolisant in Adult Patients With Idiopathic Hypersomnia

The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with idiopathic hypersomnia ...

Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE

The purpose of this study is to assess the safety, tolerability and efficacy of emapalumab in children and adults with macrophage activation syndrome (sHLH/MAS) in Still's disease (including systemic ...

Congenital Heart Disease: Impact on Learning and Development in Down Syndrome (CHILD-DS)

The study objective is to compare neurodevelopmental (ND) and behavioral outcomes between children with Down syndrome (DS) who had complete atrioventricular septal defect (CAVSD) repair and children ...

Development and Validation of Pediatric Narcolepsy Patient Reported Outcomes Scale

The purpose of this study is to test a pediatric narcolepsy patient reported outcomes tool to assess pediatric narcolepsy symptoms and their effect on daily functioning and quality of life. The goal ...

A Study of PTC923 in Participants With Phenylketonuria

The main purpose of this trial is to evaluate the efficacy of PTC923 in reducing blood phenylalanine (Phe) levels in participants with phenylketonuria as measured by mean change in blood Phe levels ...

Biomarkers of Brain Injury in Critically-Ill Children on Extracorporeal Membrane Oxygenation

The BEAM study is a multicenter, prospective, observational study in children supported on extracorporeal membrane oxygenation (ECMO). The primary goals of this study are to develop and refine a brain ...

Enhanced External Counterpulsation in Patients With Fontan Circulation

The purpose of the proposed study is to determine the safety of enhanced external counterpulsation (EECP), a system for compressing the blood vessels in the legs in synchrony with an individual's ...

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