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

Phase 1

Recruiting

• Conditions: Duchenne Muscular Dystrophy (DMD)
• Interventions: Drug: ENTR-601-44; Drug: ENTR-601-44 - matching placebo

• Registration Number: NCT07037862
• Lead Sponsor: Entrada Therapeutics, Inc.

Brief Summary

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: to evaluate if ENTR-601-44 is safe and to determine the best dose of ENTR-601-44 for Part B. 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2025-05-01
• Status Verified: 2025-06
• Study First Submitted QC: 2025-06-17
• Study First Posted: 2025-06-26
• Last Update Submitted: 2025-06-27
• Study Start: 2025-06-30
• Last Update Posted: 2025-07-01
• Primary Completion: 2029-03-28
• Study Completion: 2029-03-28

Recruitment & Eligibility

• Status: RECRUITING
• Sex: Male
• Target Recruitment: 24

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
ENTR-601-44	ENTR-601-44	intravenous infusion every 6 weeks
Placebo	ENTR-601-44 - matching placebo	intravenous infusion every 6 weeks

Primary Outcome Measures

Name	Time	Method
Number of participants with Treatment Emergent Adverse Events (TEAEs) according to study protocol (Part A)	From baseline through End of Study (up to 25 weeks).	Safety will be assessed by monitoring adverse events, physical examination, vital signs and clinical laboratory tests.

Secondary Outcome Measures

Name	Time	Method
Plasma, muscle, and urine concentration of ENTR-601-44 and its final metabolite (Part A)	From baseline through End of Study (up to 25 weeks).
Change from baseline in dystrophin by Western blot from muscle biopsy at End of Study (Part A)	Baseline, End of Study (up to 25 weeks)
Change from baseline in dystrophin expression and localization from muscle biopsy at End of Study (Part A)	Baseline, End of Study (up to 25 weeks)
Percent change from baseline in exon 44 skipping measured in muscle biopsy at End of Study (Part A)	Baseline, End of Study (up to 25 weeks)
Anti-drug antibody (ADA) and anti-dystrophin antibody in serum (Part A)	From baseline through End of Study (up to 25 weeks).

Trial Locations

Locations (14)

University Hospital Gent; 🇧🇪 Gent, Belgium

UZ Leuven; 🇧🇪 Leuven, Belgium

Centre Hospitalier Régional de la Citadelle; 🇧🇪 Liège, Belgium

IRCCS Ospedale San Raffaele; 🇮🇹 Milan, Italy

Fondazione Serena Onlus - Centro Clinico NeMO Milano; 🇮🇹 Milan, Italy

Ospedale Pediatrico Bambino Gesu; 🇮🇹 Rome, Italy

Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore; 🇮🇹 Rome, Italy

Hospital Universitario Vall d'Hebron; 🇪🇸 Barcelona, Spain

Hospital Sant Joan de Deu; 🇪🇸 Barcelona, Spain

Leeds General Infirmary; 🇬🇧 Leeds, United Kingdom

Scroll for more (4 remaining)