A 2-part, randomized, double-blind, placebo-controlled study in participants with Duchenne muscular dystrophy amenable to exon 44 skipping with an initial multiple ascending dose part A to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of ENTR-601-44, followed by Part B to evaluate the safety and efficacy of ENTR-601-44 (ELEVATE-44)
Duchenne muscular dystrophy (DMD)
Part A Experimental Arm: ENTR-601-44. • Participants will receive a fixed number of doses at one of three dose levels. One dose will be given every six weeks. • Drug: ENTR-601-44: Given by IV infusion as specified under Participant Group/Arm. Placebo Comparator Arm: ENTR-601-44 matching placebo • Participants will receive a fixed number of placebo doses matched to ENTR-601-44 doses. One dose will be given every six weeks. • Drug: ENTR-601-44 – Matching Placebo: Given by IV infusion as specified under Participant Group/Arm. Added 02/01/2026: OL Period Experimental Arm: ENTR-601-44. • Participants will receive a fixed number of doses at the dose level that was administered in their original cohort in Part A; or if they were on placebo, cross over to ENTR-601-44 at the dose level that was administered in their original cohort.
Principal inclusion criteria: 1. Genetic diagnosis of 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: 3.1. Prior treatment with any exon skipping therapy at any time 3.2. Prior treatment with any gene therapy at any time 3.3. Use of anti-coagulants, anti-thrombotics, or anti-platelet agents from at least 30 days prior to the start of the screening period until the end of the study 3.4. Use of an immunosuppressant for a non-DMD condition from 30 days prior to screening until the end of the study 3.5. Has taken or is currently taking a histone deacetylase (HDAC) inhibitor, including (but not limited to) givinostat from at least 30 days prior to the start of the screening period 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.