A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44
NCT ID: NCT07037862
Last Updated: 2025-12-15
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
24 participants
INTERVENTIONAL
2025-06-30
2029-03-28
Brief Summary
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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 be able to roll into an open-label treatment period during which the safety and efficacy of extended dosing will be evaluated.
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.
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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ENTR-601-44
intravenous infusion every 6 weeks
ENTR-601-44
intravenous infusion
Placebo
intravenous infusion every 6 weeks
ENTR-601-44 - matching placebo
intravenous infusion
Interventions
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ENTR-601-44
intravenous infusion
ENTR-601-44 - matching placebo
intravenous infusion
Eligibility Criteria
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Inclusion Criteria
2. Assigned male at birth with clinical signs compatible with Duchenne muscular dystrophy as determined by the investigator.
3. Part A: 4-20 years of age, inclusive.
4. Ambulatory Status Part A: ambulatory with a Performance of the Upper Limb v2.0 (PUL 2.0) Entry as per protocol at Screening
5. Adequate muscle for obtaining tissue biopsy as assessed by the investigator.
6. Other protocol-defined criteria apply.
Exclusion Criteria
2. 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.
3. Use of the following medications:
1. Prior treatment with any exon skipping therapy at any time
2. Prior treatment with any gene therapy at any time
3. Use of anti-coagulants, anti-thrombotics, or anti-platelet agents
4. Use of an immunosuppressants (other than oral corticosteroids for DMD conditions)
5. Has taken or is currently taking a histone deacetylase (HDAC) inhibitor, including (but not limited to) givinostat
4. Laboratory abnormalities.
5. Daytime ventilator dependence or any use of invasive mechanical ventilation via tracheostomy.
6. 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.
7. Received any experimental or investigational drug, etc. within 3 months prior to first dose or within 5 half-lives (whichever is longer).
8. Other protocol-defined criteria apply.
4 Years
20 Years
MALE
No
Sponsors
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Entrada Therapeutics, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Entrada Therapeutics Clinical Trials
Role: STUDY_DIRECTOR
Entrada Therapeutics, Inc.
Locations
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University Hospital Gent
Ghent, , 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
Alder Hey Children's NHS Foundation Trust
Liverpool, , United Kingdom
Great Ormond Street Hospital for Children
London, , United Kingdom
Royal Manchester Children's Hospital
Manchester, , United Kingdom
Freeman Hospital
Newcastle upon Tyne, , United Kingdom
Oxford University Hospitals NHS Foundation Trust
Oxford, , United Kingdom
Countries
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Facility Contacts
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Other Identifiers
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2024-517584-23-00
Identifier Type: CTIS
Identifier Source: secondary_id
U1111-1316-5469
Identifier Type: OTHER
Identifier Source: secondary_id
ENTR-601-44-201
Identifier Type: -
Identifier Source: org_study_id
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