ENTR-601-44 Exon 44 Skipping Therapy for Duchenne Muscular Dystrophy: ELEVATE-44 Clinical Trial
Actively RecruitingPhase 1/2Exon Skipping
Study Overview
- Age
- 4–20 years
- Phase
- Phase 1/2
- Sponsor
- Entrada Therapeutics
- Therapeutic Approach
- Exon Skipping
- Variant Requirement
- 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.
- Eligible Sex
- Male
- Ambulation
- Ambulatory
- Study Start (Actual)
- 2025-06-30
- Primary Completion (Estimated)
- 2029-03-28
- Study Completion (Estimated)
- 2029-03-28
- Enrollment (Estimated)
- 24
- Countries
- BelgiumItalySpainUnited Kingdom
Study Requirements and Criteria
Inclusion Criteria
- Assigned male at birth with clinical signs compatible with Duchenne muscular dystrophy as determined by the investigator.
- Part A: 4-20 years of age, inclusive.
- Ambulatory Status Part A: ambulatory with a Performance of the Upper Limb v2.0 (PUL 2.0) Entry as per protocol at Screening
- Adequate muscle for obtaining tissue biopsy as assessed by the investigator.
- Other protocol-defined criteria apply.
Exclusion Criteria
- Any significant concomitant medical condition that might interfere with the ability to comply with protocol requirements.
- 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.
- Use of the following medications:
- Prior treatment with any exon skipping therapy at any time
- Prior treatment with any gene therapy at any time
- Use of anti-coagulants, anti-thrombotics, or anti-platelet agents
- Use of an immunosuppressants (other than oral corticosteroids for DMD conditions)
- Has taken or is currently taking a histone deacetylase (HDAC) inhibitor, including (but not limited to) givinostat
- Laboratory abnormalities.
- Daytime ventilator dependence or any use of invasive mechanical ventilation via tracheostomy.
- 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.
- Received any experimental or investigational drug, etc. within 3 months prior to first dose or within 5 half-lives (whichever is longer).
- Other protocol-defined criteria apply.
Clinical Trial Registry
NCT ID
NCT07037862This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.