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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A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat ...
The primary objective of the 1701-202 STRONG SCD study is to evaluate the safety and tolerability of different dose levels of IW-1701 compared with placebo when administered daily for approximately 12 ...
The purpose of this study is to assess AR101's safety, tolerability and efficacy over an extended dosing period.
This study evaluates the ability of contrast-enhanced ultrasound to improve the diagnosis of acute appendicitis in children compared to conventional ultrasound.
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that ...
Phase 3 pivotal US trial studying open-label intravenous administration of onasemnogene abeparvovec-xioi in spinal muscular atrophy (SMA) Type 1 participants.
This is a single center, prospective, observational study to demonstrate the clinical validity of the Intrinsic LifeSciences (ILS) Intrinsic Hepcidin IDx™ Test in the diagnosis and management of iron ...
This is a phase I/II open label multi-center study in which patients will receive low dose targeted busulfan followed by infusion of autologous CD34+ selected bone marrow or mobilized peripheral blood ...
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 ...
131I-Metaiodobenzylguanidine (131I-MIBG) is one of the most effective therapies utilized for neuroblastoma patients with refractory or relapsed disease. In this pediatric phase 1 trial, 131I-MIBG will ...