At Boston Children’s Hospital, clinical trials help us discover new and better ways to care for children. These studies give families the chance to explore promising treatments, especially when other options haven’t worked. Participation is always voluntary, and our team is here to guide you, answer questions, and support you throughout the process.
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The main objective of this study is to evaluate the efficacy of SRP-4045 (casimersen) and SRP-4053 (golodirsen) compared to placebo in participants with DMD with out-of-frame deletion mutations ...
This Registry will enroll adolescent and pediatric participants who received at least one Gilead Hepatitis C Virus (HCV) direct acting antiviral (DAA) while participating in a Gilead-sponsored chronic ...
Children undergoing hematopoietic cell transplantation (HCT) for cancer or blood disorders frequently develop gastrointestinal, metabolic and infectious complications related to preparative high-dose ...
This research study is evaluating the use of specialized testing of solid tumors including sequencing. The process of performing these specialized tests is called tumor profiling. The tumor profiling ...
Due to different study designs, the sponsor separated Part C into a separate registration (NCT04958642), leaving Parts A/B here in NCT02534844. This study is to find out how safe and effective VTS ...
This is a longitudinal, observational study of patients with Hemophilia A or B who are planning to switch to a newly approved coagulation factor replacement product, or who have recently switched ...
The goal is to determine how lifestyle and exercise impact the well-being of individuals with hypertrophic cardiomyopathy (HCM) and long QT syndrome (LQTS). Ancillary study Aim: To understand how the ...
This study will determine if air-displacement plethysmography (ADP) provides accurate measurement of body composition (percent body fat and fat-free mass) in pediatric patients with intestinal failure ...
The purpose of this study is to evaluate the effects of early adenotonsillectomy (eAT) on the behavior, sleep-disordered breathing symptoms and quality of life for children who snore, but do not have ...
This randomized, double-blind, controlled, outpatient two-period, two-treatment crossover study is designed to evaluate the efficacy and safety of amifampridine phosphate in patients (ages 2 and above ...