Actively Recruiting

Phase 1
Phase 2
Age: 4Years - 20Years
MALE
NCT07037862

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

Led by Entrada Therapeutics, Inc. · Updated on 2026-03-09

24

Participants Needed

14

Research Sites

195 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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

Official Title

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

Who Can Participate

Age: 4Years - 20Years
MALE

Eligibility Criteria

Eligible

You may qualify if you...

  • Genetic diagnosis of Duchenne muscular dystrophy (DMD) with a confirmed variant amenable to exon 44 skipping
  • Assigned male at birth with clinical signs of DMD
  • Aged 4 to 20 years inclusive for Part A
  • Ambulatory with Performance of the Upper Limb v2.0 entry at screening
  • Adequate muscle tissue available for biopsy
  • Other protocol-defined criteria apply
Not Eligible

You will not qualify if you...

  • Any significant medical condition affecting ability to follow study requirements
  • Acute illness within 4 weeks before first dose that may interfere with safety or measurements
  • Prior treatment with exon skipping therapy or gene therapy at any time
  • Use of anti-coagulants, anti-thrombotics, or anti-platelet agents
  • Use of immunosuppressants other than oral corticosteroids for DMD
  • Has taken or is currently taking histone deacetylase inhibitors, including givinostat
  • Laboratory abnormalities
  • Daytime ventilator dependence or invasive mechanical ventilation via tracheostomy
  • Abnormal ECG or QTcF interval >450 msec at screening or before first dose
  • Received experimental drugs within 3 months or 5 half-lives before first dose
  • Other protocol-defined criteria apply

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

1
2
3
+1

Trial Site Locations

Total: 14 locations

1

University Hospital Gent

Ghent, Belgium, 9000

Actively Recruiting

2

UZ Leuven

Leuven, Belgium, 3000

Actively Recruiting

3

Centre Hospitalier Régional de la Citadelle

Liège, Belgium, 4000

Actively Recruiting

4

IRCCS Ospedale San Raffaele

Milan, Italy, 20132

Actively Recruiting

5

Fondazione Serena Onlus - Centro Clinico NeMO Milano

Milan, Italy, 20162

Actively Recruiting

6

Ospedale Pediatrico Bambino Gesu

Rome, Italy, 00165

Actively Recruiting

7

Hospital Universitario Vall d'Hebron

Barcelona, Spain, 08035

Actively Recruiting

8

Hospital Sant Joan de Deu

Barcelona, Spain, 08950

Actively Recruiting

9

Leeds General Infirmary

Leeds, United Kingdom, LS1 3EX

Actively Recruiting

10

Alder Hey Children's NHS Foundation Trust

Liverpool, United Kingdom, L122AP

Not Yet Recruiting

11

Great Ormond Street Hospital for Children

London, United Kingdom, WC1N 3JH

Actively Recruiting

12

Royal Manchester Children's Hospital

Manchester, United Kingdom, M13 9WL

Not Yet Recruiting

13

Freeman Hospital

Newcastle upon Tyne, United Kingdom, NE1 3BZ

Actively Recruiting

14

Oxford University Hospitals NHS Foundation Trust

Oxford, United Kingdom, OX3 9DU

Actively Recruiting

Loading map...

How is the study designed?

Study Type

INTERVENTIONAL

Masking

QUADRUPLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

Not the Right Trial for You?

Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.

Already have an account? Log in here