NCT05524883

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 86 (actual)
• Sponsor: Dyne Therapeutics · Industry
• Sex: Male
• Age: 4 Years – 16 Years
• Healthy volunteers: Not accepted

Summary

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 (LTE) period (192 weeks).

Conditions

• Duchenne Muscular Dystrophy (DMD)

Interventions

Type	Name	Description
DRUG	DYNE-251	Administered by IV infusion
DRUG	Placebo	Administered by IV infusion

Timeline

• Start date: 2022-08-12
• Primary completion: 2029-11-01
• Completion: 2029-11-01
• First posted: 2022-09-01
• Last updated: 2025-08-13

Locations

30 sites across 9 countries: United States, Australia, Belgium, Canada, Ireland, Italy, South Korea, Spain, United Kingdom

Regulatory

• FDA-regulated drug study