Actively Recruiting
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
Eligibility Criteria
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
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
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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
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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