A 2-Part, Randomized, Double-Blind, Placebo-Controlled Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy of ENTR-601-45

What this Trial Is About

Researchers are evaluating the investigational medicine ENTR-601-45 in males aged 4 to 20 years with Duchenne muscular dystrophy (DMD), a rare genetic condition affecting muscle function. The study aims to assess the safety and potential positive effects of ENTR-601-45 compared to a placebo, which looks like the study drug but contains no active ingredient. This trial consists of two parts: Part A focuses on safety and dose determination, while Part B further evaluates safety and effectiveness at the chosen dose. Participants may also join an open-label period for extended safety and efficacy assessment. Participants will receive multiple intravenous (IV) infusions of ENTR-601-45 or placebo every six weeks during Parts A and B. The open-label period allows participants to receive the study treatment with ongoing monitoring. Throughout the study, participants will continue their standard care for DMD if their health remains stable. The study involves regular clinic visits for treatment and evaluations. During the trial, participants will undergo various tests including blood and urine analyses, physical exams, questionnaires, muscle biopsies at the start and end of treatment, and exercise tests. Researchers will monitor treatment-emergent side effects, drug levels in the body, dystrophin protein changes in muscle tissue, exon 45 skipping effects, antibody responses, and physical function measures such as walking and stair climbing. The study may last up to 62 weeks, with close safety monitoring throughout and after treatment.

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45