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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Lorlatinib is a novel inhibitor across ALK variants, including those resistant to crizotinib. In this first pediatric phase 1 trial of lorlatinib, the drug will be utilized as a single agent and in ...
This is a Phase 3 study to assess the safety and efficacy of Tadekinig alfa in patients with monogenic, interleukin-18 (IL 18) driven autoinflammation due to Nucleotide-binding oligomerization domain, ...
This study will evaluate the efficacy and safety of OC5 in patients with PH.
The goal of the Fontan Imaging Biomarkers (FIB) study is to identify the associations of blood and urine biomarkers to imaging parameters of ventricular mechanics.
A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for F508del.
Infants often present to the hospital with episodes of coughing, choking, gagging, change in muscle tone, and/or change in skin color, known as brief resolved unexplained event. Many studies have ...
This study will explore whether ivabradine lowers heart rate, and thus improves exercise capacity, in survivors of lymphoma who have an elevated resting heart rate as a side effect of prior radiation ...
The purpose of this study is to evaluate vedolizumab pharmacokinetics (PK), safety and tolerability in pediatric participants with moderately to severely active UC or CD.
Cerebrovascular events, such as stroke, are a devastating complication of Fabry disease that results in part from storage of complex lipids in both large and small vessels. Understanding how the ...
SMART T1D is a research study that offers interactive diabetes education for teens with type 1 diabetes in peer groups led by diabetes nurse educators. Participants in the study will be randomly ...