NCT06415344
Long-term Extension of GTX-102 in Angelman Syndrome
The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)
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The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)
Despite the increasingly common use of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies in treating cystic fibrosis (CF), it is still largely unknown whether or not other ...
Despite the increasingly common use of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies in treating cystic fibrosis (CF), it is still largely unknown whether or not other ...
This is a prospective, randomized, controlled, non-inferiority study of patients undergoing tonsil surgeries at Boston Children's Hospital Waltham. The overall aim is to evaluate the efficacy of an ...
The purpose of this study is to evaluate the efficacy of SPK-8011 in preventing bleed episodes compared with FVIII prophylaxis in participants with hemophilia A without FVIII inhibitors on routine ...
The goal of this clinical trial is to learn about the feasibility and acceptability of a behavioral intervention in designed to provide sexual education and improve the behavioral skills of autistic ...
The goal of this translational biomarker study is to use electroencephalography (EEG) to identify brain signatures that will predict a child's response to two of the most commonly prescribed ADHD ...
This study aims to learn more about blood management in children undergoing heart surgery, such as the right amounts, and the best blood products, to administer. It also aims to develop a mathematical ...
a 32-week study that will evaluate the safety, tolerability and preliminary efficacy of multiple ascending doses of crofelemer, compared to placebo, using a randomized cross-over design within each ...
This research is being done to test a new drug called PEEL-224 in combination with two commercially available drugs, Vincristine and Temozolomide, and to determine how effective this combination of ...