ENTR-601-44 treatment for Duchenne muscular dystrophy eligible for exon 44 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 interventional study enrolls males assigned at birth aged 4–20 with genetically confirmed DMD amenable to exon 44 skipping. Part A is a double-blind, dose-finding period comparing ENTR-601-44 to a matching placebo with an open-label extension for longer-term safety and dosing; Part B further studies safety and effects at the selected dose. Participants receive multiple IV doses, undergo muscle biopsy and regular safety and functional assessments, and are monitored for side effects and potential increases in dystrophin or functional change. The goal is to identify a tolerable dosing regimen and look for signs of clinical or biomarker benefit versus placebo.

Why it matters

Who can join: the full rules

Principal inclusion criteria

1. Genetic diagnosis of Duchenne muscular dystrophy (DMD) and confirmed pathologic variant in the dystrophin gene amenable to exon 44 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.

Principal 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 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.

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
• Fondazione Serena Onlus - Centro Clinico NeMO Milano
  Milan, Italy Recruiting
• Ospedale Pediatrico Bambino Gesu
  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.