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

Incidence and severity of treatment emergent adverse events (TEAEs) (Part A and OL Period)), Changes in vital sign measurements (Part A and OL Period), Changes in clinical laboratory results (Part A and OL Period), Changes in electrocardiogram (ECG) parameters (Part A and OL Period), Changes in physical examination findings (Part A and OL Period)

Plasma, muscle, and urine concentration of ENTR-601-44 and its final metabolite (Part A and OL Period), Change from baseline in dystrophin by Western blot from muscle biopsy (Part A), Change from baseline to End of Part A in dystrophin expression and localization from muscle biopsy (Part A), Percent change from baseline to End of Part A in exon 44 skipping measured in muscle biopsy (Part A), Anti-drug antibody (ADA) and anti-dystrophin antibody in serum (Part A and OL Period)), Change from baseline to End of OL Period in 10-Meter Walk/Run (10MWR) (Part A and OL Period), Change from baseline to End of OL Period in Timed Rise from Floor (Part A and OL Period), Change from baseline to End of OL Period in Timed 4-Stair Climb (4SC) (Part A and OL Period), Change from baseline to End of OL Period in 95th centile Stride Velocity (SV95C) (Part A and OL Period), Change from baseline to End of OL Period in North Star Ambulatory Assessment (NSAA) (Part A and OL Period), Change from baseline to End of OL Period in Performance of the Upper Limb v2.0 (PUL 2.0) (Part A and OL Period)

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