DYNE-251 Exon 51 Skipping Therapy for Duchenne Muscular Dystrophy: FORZETTO Clinical Trial
Actively RecruitingPhase 3Exon Skipping
Study Overview
- Age
- 4–18 years
- Phase
- Phase 3
- Sponsor
- Dyne Therapeutics
- Therapeutic Approach
- Exon Skipping
- Variant Requirement
- Ambulatory male with confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping.
- Eligible Sex
- Male
- Ambulation
- Ambulatory
- Study Start (Actual)
- 2026-06
- Primary Completion (Estimated)
- 2030-12
- Study Completion (Estimated)
- 2032-10
- Enrollment (Estimated)
- 90
- Countries
- United States
Study Requirements and Criteria
Steroid Use
Receiving a stable daily or weekend dosage of glucocorticoids for at least 24 weeks prior to randomization with the expectation of maintaining a stable dose during the Placebo-Controlled Period of the study (unless dose adjustment is required by weight change)
Inclusion Criteria
Rise From Floor (RFF) time must be < 10 seconds for both screening assessments.
Exclusion Criteria
- Receipt of ongoing immunosuppressive therapy (other than glucocorticoids) within 12 weeks prior to randomization
- Use of any pharmacologic treatment (other than glucocorticoids) that may have an effect on muscle strength or function within 12 weeks prior to randomization
- Any change in prophylaxis/treatment for congestive heart failure (CHF) within 12 weeks prior to randomization
- Receipt of eteplirsen within 1 week prior to randomization
- Receipt of alternative exon-skipping or dystrophin-modifying therapy or zeleciment rostudirsen within 24 weeks prior to randomization
- Receipt of givinostat within 12 weeks prior to randomization
- Receipt of gene therapy at any time
- Note: Other inclusion or exclusion criteria may apply
Contact Information
This section provides contact details for people who can answer questions about joining this study
- Name: Dyne Clinical Trials
- Phone Number: +1-781-317-1919
- Email: [email protected]
Clinical Trial Registry
NCT ID
NCT07608432This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.