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

Launched by ENTRADA THERAPEUTICS, INC. · Jun 17, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new medicine called ENTR-601-44 for boys and young men with Duchenne muscular dystrophy (DMD), a rare genetic condition that weakens muscles over time. The goal is to see if this medicine is safe to use, to learn about any side effects, and to find out if it helps improve muscle health compared to a placebo, which is a treatment that looks like the medicine but doesn’t have any active ingredients.

Boys and young men aged 4 to 20 who have a confirmed diagnosis of DMD caused by a specific genetic change called “exon 44 skipping” may be eligible to join. Participants need to be able to walk and have enough muscle for doctors to take small tissue samples (biopsies) from their muscles. During the study, participants will receive the study treatment through slow injections into a vein over several weeks and will visit the clinic regularly for checkups, blood and urine tests, questionnaires, and exercise tests. They will have muscle biopsies at the start and end of the study to see if the treatment made any difference. Participants can continue their usual DMD care as long as their health stays stable. This study is not yet recruiting but aims to help find new treatment options for people with this form of DMD.

Gender

MALE

Eligibility criteria

• • Principal inclusion criteria
• • 1. Genetic diagnosis of DMD and confirmed pathologic variant in the dystrophin gene amenable to exon 44 skipping as reviewed by a central genetic counselor.
• • 2. Assigned male at birth with clinical signs compatible with Duchenne muscular dystrophy as determined by the investigator.
• • 3. Part A: 4-20 years of age, inclusive.
• • 4. Ambulatory Status Part A: ambulatory with a Performance of the Upper Limb v2.0 (PUL 2.0) Entry as per protocol at Screening
• • 5. Adequate muscle for obtaining tissue biopsy as assessed by the investigator.
• • 6. Other protocol-defined criteria apply.
• • Principal exclusion criteria
• • 1. Any significant concomitant medical condition that might interfere with the ability to comply with protocol requirements.
• • 2. Has an acute illness within 4 weeks prior to the first dose of study drug which may interfere with study measurements or jeopardize participant's safety.
• 3. Use of the following medications:
• • 1. Prior treatment with any exon skipping therapy at any time
• • 2. Prior treatment with any gene therapy at any time
• • 3. Use of anti-coagulants, anti-thrombotics, or anti-platelet agents from at least 30 days prior to the start of the screening period until the end of the study
• • 4. Use of an immunosuppressant for a non-DMD condition from 30 days prior to screening until the end of the study
• • 5. Has taken or is currently taking a histone deacetylase (HDAC) inhibitor, including (but not limited to) givinostat from at least 30 days prior to the start of the screening period until the end of the study
• • 4. Laboratory abnormalities.
• • 5. Daytime ventilator dependence or any use of invasive mechanical ventilation via tracheostomy.
• • 6. Has an abnormal electrocardiogram (ECG) reading assessed as clinically significant by the investigator, and/or a QT interval with Fridericia correction method (QTcF) \>450 msec at Screening or prior to the first dose of study drug on Day 1.
• • 7. Received any experimental or investigational drug, etc. within 3 months prior to first dose or within 5 half-lives (whichever is longer).
• • 8. Other protocol-defined criteria apply.