Study of DYNE-251 for Duchenne Muscular Dystrophy amenable to Exon 51 Skipping

Dyne Therapeutics is recruiting patients for the clinical trial of Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (DELIVER).

The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled period (24 weeks), an open-label period (24 weeks) and a long-term extension period (96 weeks).

The researchers plan that August 12, 2022 will be the study start date. The indicative completion of the clinical trial will be expected in November 2026.

Among primary outcome measures are the Change From Baseline in Dystrophin Protein Levels in Muscle Tissue at Week 25.

The page dedicated to this clinical trial can be found here: https://ichgcp.net/clinical-trials-registry/NCT05524883.