ENTR-601-45 for Duchenne muscular dystrophy patients amenable to exon 45 skipping

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 1/2 program uses a two-part design with an initial dose-finding/safety Part A followed by a controlled Part B at the selected dose. Participants receive multiple IV infusions of ENTR-601-45 or matching placebo, with safety, side effects, and signs of benefit compared against placebo in Part B. Those completing the randomized phases may roll into an open-label extension to assess longer-term safety and continued dosing effects. Genetic eligibility is centrally confirmed and prior exon-skipping therapy is excluded.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Genetic diagnosis of DMD and confirmed pathologic variant in the dystrophin gene amenable to exon 45 skipping as reviewed by a central genetic counselor.
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:

1. Any significant concomitant medical condition that might interfere with the ability to comply with protocol requirements.
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 or current treatment with any exon skipping therapy within the previous 12 months
   2. Prior or current treatment with any gene therapy
   3. Use of anti-coagulants, anti-thrombotics, or anti-platelet agents from 30 days prior to screening and until the end of the study
   4. Use of an immunosuppressant (other than systemic or oral corticosteroid for DMD condition) from 30 days prior to screening until the end of the study.
   5. Treatment with a histone deacetylase (HDAC) inhibitor, including (but not limited to) givinostat from 30 days prior to screening until the end of the study
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.

Where it's running

• University Hospital Gent
  Ghent, Belgium Recruiting
• UZ Leuven
  Leuven, Belgium Recruiting
• Centre Hospitalier Régional de la Citadelle
  Liège, Belgium Recruiting
• IRCCS Ospedale San Raffaele
  Milan, Italy Recruiting
• Ospedale Pediatrico Bambino Gesu
  Rome, Italy Recruiting
• Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
  Rome, Italy Recruiting

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.