A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44
Recruiting now · Phase 1/Phase 2 · Has a placebo group
Conditions studied: Duchenne Muscular Dystrophy (DMD)
In brief
This is a study of the investigational medicine ENTR-601-44 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition. The researchers want to: Test how safe ENTR-601-44 is, learn about any side effects, and look at the potential positive effects of ENTR-601-44, compared to placebo. Placebo looks like the investigational medicine but does not contain any active ingredient. In this summary ENTR-601-44 and placebo are both called study treatments. The study has 2 parts: * Part A * A Double-Blind Period, to evaluate if ENTR-601-44 is safe and to determine the best dose of ENTR-601-44 for Part B. * Following the Double-Blind period, participants will roll into an open-label treatment period during which the safety and efficacy of extended dosing will be evaluated. * Part B * To further evaluate the effect and safety of ENTR-601-44 at the dose determined in Part A. Participants will: * Receive study treatment in the form of multiple intravenous (IV) infusions (slow injection) into a vein over the course of several weeks in Part A and in Part B * Visit the clinic regularly for checkups and tests such as: blood and urine tests, physical examinations, questionnaires, and exercise tests. Participants will have a muscle biopsy at the beginning of their participation and after their last dose to allow researchers to compare whether there have been changes in the muscle as a result of the study drug. Participants are allowed to continue receiving their standard of care therapy for DMD during the study, as long as their health remains stable.
Key facts
- Study ID
- NCT07037862
- Run by
- Entrada Therapeutics, Inc.
- People needed
- 24
- Starts
- 2025-06-30
- Expected to finish
- 2029-03-28
- Last updated by the study team
- 2026-03-09
Who can join
Age: 4 and older, up to 20. Sex: male. Healthy volunteers: not accepted.
Where it is running
- Oxford University Hospitals NHS Foundation Trust — Oxford, United Kingdom (enrolling)
- UZ Leuven — Leuven, Belgium (enrolling)
- Centre Hospitalier Régional de la Citadelle — Liège, Belgium (enrolling)
- IRCCS Ospedale San Raffaele — Milan, Italy (enrolling)
- Fondazione Serena Onlus - Centro Clinico NeMO Milano — Milan, Italy (enrolling)
- Ospedale Pediatrico Bambino Gesu — Rome, Italy (enrolling)
- Hospital Universitario Vall d'Hebron — Barcelona, Spain (enrolling)
- Hospital Sant Joan de Deu — Barcelona, Spain (enrolling)
- Leeds General Infirmary — Leeds, United Kingdom (enrolling)
- Freeman Hospital — Newcastle upon Tyne, United Kingdom (enrolling)
- University Hospital Gent — Ghent, Belgium (enrolling)
- Great Ormond Street Hospital for Children — London, United Kingdom (enrolling)
- Alder Hey Children's NHS Foundation Trust — Liverpool, United Kingdom
- Royal Manchester Children's Hospital — Manchester, United Kingdom
Full record on ClinicalTrials.gov
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