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FORZETTO: DYNE-251 in Duchenne Muscular Dystrophy Amenable to Exon-51 Skipping
NCT07608432Phase 3
Key facts
- Status
- Recruiting
- Start
- Jun 2026
- Primary completion
- Dec 2030
- Countries
- 1
- Sponsor
- Dyne Therapeutics
- Conditions
- Duchenne muscular dystrophy (DMD)
01
Overview
Phase 3, randomised, double-blind trial in Duchenne muscular dystrophy (DMD) comparing Zeleciment Rostudirsen (DYNE-251) with Placebo (2 arms). Primary endpoint: Rise From Floor velocity at Week 73. Started Jun 2026; status: recruiting.
02
Design
2 arms- Study design
- RandomizedDouble-blind
Zeleciment Rostudirsen (DYNE-251)
Participants receive zeleciment rostudirsen Q4W for 72 weeks, then continue zeleciment rostudirsen Q4W for 96 weeks.
Placebo
Participants receive placebo Q4W for 72 weeks, then switch to zeleciment rostudirsen Q4W for 96 weeks.
03
Who it enrols
Ambulatory males 4-18 years old with DMD amenable to exon 51 skipping, on stable glucocorticoids, with RFF time < 10 seconds.
04
Endpoints
Primary
Rise From Floor velocity
Secondary
Rise From Floor velocity
Stride Velocity 95th Percentile
North Star Ambulatory Assessment Total Score
10-Meter Walk/Run Velocity
4-Stair Climb velocity
Functional Composite score
Forced Vital Capacity
Patient Global Impression of Severity
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