ENTR-601-44 Exon 44 Skipping Therapy for Duchenne Muscular Dystrophy: ELEVATE-44 Clinical Trial

Actively RecruitingPhase 1/2Exon Skipping

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

Not yet provided

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.

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.