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

NCT07037862 | Recruiting Phase 1 DMC FDA Drug

• 3 other identifiers: ENTR-601-44-2012024-517584-23-00U1111-1316-5469
• Study Type: interventional
• Target: 24
• Locations: 4 countries
• Sites: 14

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
• 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.

Conditions

Duchenne Muscular Dystrophy (DMD)

Keywords

Duchenne Muscular Dystrophy; DMD; exon skipping therapy; oligonucleotide therapy

Outcome Measures

Primary Outcomes (1)

• Number of participants with Treatment Emergent Adverse Events (TEAEs) according to study protocol (Part A and Open Label (OL) Period)

  Safety will be assessed by monitoring adverse events, physical examination, vital signs and clinical laboratory tests.

  From baseline through End of Study (up to 62 weeks).

Secondary Outcomes (11)

• Plasma, muscle, and urine concentration of ENTR-601-44 and its final metabolite (Part A and Open Label (OL) Period)

  From Baseline through End of Study (up to 62 weeks).

• Change from baseline to End of Part A in dystrophin by Western blot from muscle biopsy (Part A)

  Baseline, End of Part A (up to 25 weeks)

• Change from baseline to End of Part A in dystrophin expression and localization from muscle biopsy (Part A)

  Baseline, End of Part A (up to 25 weeks)

• Percent change from baseline to End of Part A in exon 44 skipping measured in muscle biopsy at End of Study (Part A)

  Baseline, End of Part A (up to 25 weeks)

• Anti-drug antibody (ADA) and anti-dystrophin antibody in serum (Part A and OL Period)

  From baseline through End of Study (up to 62 weeks).

Study Arms (2)

ENTR-601-44

EXPERIMENTAL

intravenous infusion every 6 weeks

Drug: ENTR-601-44

Placebo

PLACEBO COMPARATOR

intravenous infusion every 6 weeks

Drug: ENTR-601-44 - matching placebo

Interventions

ENTR-601-44 DRUG

intravenous infusion

ENTR-601-44

ENTR-601-44 - matching placebo DRUG

intravenous infusion

Placebo

Eligibility Criteria

• Age: 4 Years - 20 Years
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Genetic diagnosis of Duchenne muscular dystrophy (DMD) and confirmed pathologic variant in the dystrophin gene amenable to exon 44 skipping as reviewed by a central genetic counselor.
• Assigned male at birth with clinical signs compatible with Duchenne muscular dystrophy as determined by the investigator.
• Part A: 4-20 years of age, inclusive.
• Ambulatory Status Part A: ambulatory with a Performance of the Upper Limb v2.0 (PUL 2.0) Entry as per protocol at Screening
• Adequate muscle for obtaining tissue biopsy as assessed by the investigator.
• Other protocol-defined criteria apply.

You may not qualify if:

• Any significant concomitant medical condition that might interfere with the ability to comply with protocol requirements.
• 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.
• Use of the following medications:
• Prior treatment with any exon skipping therapy at any time
• Prior treatment with any gene therapy at any time
• Use of anti-coagulants, anti-thrombotics, or anti-platelet agents
• Use of an immunosuppressants (other than oral corticosteroids for DMD conditions)
• Has taken or is currently taking a histone deacetylase (HDAC) inhibitor, including (but not limited to) givinostat
• Laboratory abnormalities.
• Daytime ventilator dependence or any use of invasive mechanical ventilation via tracheostomy.
• 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.
• Received any experimental or investigational drug, etc. within 3 months prior to first dose or within 5 half-lives (whichever is longer).
• Other protocol-defined criteria apply.

Sponsors & Collaborators

• Entrada Therapeutics, Inc.: lead

Study Sites (14)

University Hospital Gent

Ghent, 9000, Belgium

RECRUITING

UZ Leuven

Leuven, 3000, Belgium

RECRUITING

Centre Hospitalier Régional de la Citadelle

Liège, 4000, Belgium

RECRUITING

IRCCS Ospedale San Raffaele

Milan, 20132, Italy

RECRUITING

Fondazione Serena Onlus - Centro Clinico NeMO Milano

Milan, 20162, Italy

RECRUITING

Ospedale Pediatrico Bambino Gesu

Rome, 00165, Italy

RECRUITING

Hospital Universitario Vall d'Hebron

Barcelona, 08035, Spain

RECRUITING

Hospital Sant Joan de Deu

Barcelona, 08950, Spain

RECRUITING

Leeds General Infirmary

Leeds, LS1 3EX, United Kingdom

RECRUITING

Alder Hey Children's NHS Foundation Trust

Liverpool, L122AP, United Kingdom

NOT YET RECRUITING

Great Ormond Street Hospital for Children

London, WC1N 3JH, United Kingdom

RECRUITING

Royal Manchester Children's Hospital

Manchester, M13 9WL, United Kingdom

NOT YET RECRUITING

Freeman Hospital

Newcastle upon Tyne, NE1 3BZ, United Kingdom

RECRUITING

Oxford University Hospitals NHS Foundation Trust

Oxford, OX3 9DU, United Kingdom

RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Entrada Therapeutics Clinical Trials

  Entrada Therapeutics, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 1, 2025
• First Posted: June 26, 2025
• Study Start: June 30, 2025
• Primary Completion (Estimated): March 28, 2029
• Study Completion (Estimated): March 28, 2029
• Last Updated: March 9, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

IT (3); ES (2); GB (6); BE (3)