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Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of DYNE-251 Administered Intravenously in Ambulatory Male Participants 4 to 18 Years of Age With Duchenne Muscular Dystrophy Amenable to Exon-51 Skipping
Who can join
Ages 4 to 18 · Men
Full eligibility criteria
Inclusion Criteria: * Ambulatory male with confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping . * Rise From Floor (RFF) time must be \< 10 seconds for both screening assessments . * 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) 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
About the study
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18 years of age, with dystrophin mutations amenable to exon 51 skipping.
What is being tested
- Zeleciment Rostudirsen (DYNE-251) (drug)
- Placebo (drug)
Sponsor: Dyne Therapeutics · Participants: 90 · Started: 2026-06
Contact the study team
- Dyne Clinical Trials · Phone: +1-781-317-1919
Official record on ClinicalTrials.gov — NCT07608432
Locations in the U.S.
| North Carolina | Rare Disease Research, LLC, Hillsborough |
Conditions
- Duchenne Muscular Dystrophy
- Muscular Dystrophy
- Myotonic Dystrophy
- Genetic Disorders
- X-Linked Genetic Diseases
- and Neonatal Diseases and Abnormalities Hereditary Congenital
- Neuromuscular Disorders
From ClinicalTrials.gov, data retrieved Sep 29, 2026. Each study sets its own eligibility; the study team decides who can join.