Actively Recruiting
A 2-Part, Randomized, Double-Blind, Placebo-Controlled Study in Males Aged 4-20 Years 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
N/A
Total Duration
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AI-Summary
What this Trial Is About
Researchers are evaluating the investigational medicine ENTR-601-44 in males aged 4 to 20 years with Duchenne muscular dystrophy (DMD), a rare genetic condition affecting muscles. The study aims to test the safety, side effects, and potential positive effects of ENTR-601-44 compared to a placebo. It is a two-part, randomized, double-blind, placebo-controlled trial with an initial dose-finding phase followed by an extended treatment phase. Participants will receive multiple intravenous infusions of ENTR-601-44 or placebo every six weeks during both parts of the study. Part A includes a double-blind period to assess safety and find the best dose, followed by an open-label period to evaluate extended dosing effects. Part B further evaluates the safety and efficacy of the selected dose. Participants may continue their standard DMD therapy if their health is stable. Throughout the study, participants will have regular clinic visits for blood and urine tests, physical exams, questionnaires, and exercise tests. Muscle biopsies will be taken at the start and after the last dose to assess changes in muscle tissue. Researchers will monitor adverse events, drug levels in plasma, muscle, and urine, and several measures of muscle function and mobility. The study may last up to 62 weeks, including follow-up and open-label treatment.
CONDITIONS
Brief 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 amenable to exon 44 skipping confirmed by central genetic counselor
- Assigned male at birth with clinical signs compatible with Duchenne muscular dystrophy
- Aged between 4 and 20 years inclusive (Part A)
- Ambulatory with Performance of the Upper Limb v2.0 (PUL 2.0) at screening
- Adequate muscle tissue for biopsy as assessed by investigator
- Other protocol-defined criteria apply
You will not qualify if you...
- Significant medical conditions interfering with protocol compliance
- Acute illness within 4 weeks before first dose that may affect measurements or safety
- Prior treatment with exon skipping or gene therapy
- Use of anti-coagulants, anti-thrombotics, anti-platelet agents, or immunosuppressants other than oral corticosteroids for DMD
- Use or history of histone deacetylase (HDAC) inhibitors, including givinostat
- Laboratory abnormalities
- Daytime ventilator dependence or use of invasive mechanical ventilation
- Abnormal ECG with QTcF >450 msec or clinically significant findings
- Receipt of experimental or investigational drug 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
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 25 weeks
Participants receive intravenous infusions of ENTR-601-44 or matching placebo every 6 weeks as part of the treatment phase.
Infusions every 6 weeks during treatment
Duration - Up to 37 weeks following treatment (up to 62 weeks total study duration)
Participants are monitored for safety and efficacy outcomes following the treatment phase, including assessments of muscle function and antibody levels.
Periodic visits up to study end
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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