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NCT07608432PHASE3Recruiting

Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)

Dyne Therapeutics

Start Date

6/1/2026

Completion Date

10/1/2032

Summary

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.

Detailed Description

The study consists of three periods: a Screening period (up to 6 weeks), a Placebo-Controlled Period (72 weeks) and an open-label Long-Term Extension Period (96 weeks).

Eligibility Criteria

Age Range: 4 years to 18 years

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

Interventions

DRUG

Zeleciment Rostudirsen (DYNE-251)

DRUG

Placebo

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Conditions

Duchenne Muscular Dystrophy (DMD)Muscular Dystrophy, DuchenneMuscular Dystrophy (DMD)DMDMuscular DystrophiesMuscular Dystrophy in ChildrenMuscular Dystrophy, Duchenne TypeMuscular Dystrophy, Duchenne and Becker TypesMuscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)Genetic Disease, InbornGenetic Disease, X-LinkedCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNeuromuscular Diseases (NMD)

Locations

Rare Disease Research, LLC

Hillsborough, North Carolina 27278

United States