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

Recruiting now · Phase 3 · Has a placebo group

Conditions studied: Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne, Muscular Dystrophy (DMD), DMD, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Duchenne Type, Muscular Dystrophy, Duchenne and Becker Types, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Genetic Disease, Inborn, Genetic Disease, X-Linked, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neuromuscular Diseases (NMD)

In brief

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.

Key facts

Study ID

NCT07608432

Run by

Dyne Therapeutics

People needed

90

Starts

2026-06-01

Expected to finish

2032-10-01

Last updated by the study team

2026-05-27

Who can join

Age: 4 and older, up to 18. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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)

You may not qualify if…

• 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

Where it is running

• Rare Disease Research, LLC — Hillsborough, North Carolina, United States (enrolling)

Full record on ClinicalTrials.gov

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