Study Evaluating the Safety and Effectiveness of ENTR-601-44 in Boys Aged 4 to 20 with Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping

What this Trial Is About

Researchers are evaluating the investigational medicine ENTR-601-44 in males aged 4 to 20 years with Duchenne muscular dystrophy DMD, a rare genetic condition affecting muscles. The study aims to test the safety, side effects, and potential positive effects of ENTR-601-44 compared to a placebo. It is a two-part, randomized, double-blind, placebo-controlled trial with an initial dose-finding phase followed by an extended treatment phase. Participants will receive multiple intravenous infusions of ENTR-601-44 or placebo every six weeks during both parts of the study. Part A includes a double-blind period to assess safety and find the best dose, followed by an open-label period to evaluate extended dosing effects. Part B further evaluates the safety and efficacy of the selected dose. Participants may continue their standard DMD therapy if their health is stable. Throughout the study, participants will have regular clinic visits for blood and urine tests, physical exams, questionnaires, and exercise tests. Muscle biopsies will be taken at the start and after the last dose to assess changes in muscle tissue. Researchers will monitor adverse events, drug levels in plasma, muscle, and urine, and several measures of muscle function and mobility. The study may last up to 62 weeks, including follow-up and open-label treatment.

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44