Health Trials

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

ConditionsDuchenne 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)
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexMale
Age4-18
SponsorDyne Therapeutics

About this trial

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.

Eligibility criteria

Qualifiers

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)

Disqualifiers

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

Trial design

Treatments tested in this trial

  • Zeleciment Rostudirsen (DYNE-251)
  • Placebo

Treatment groups

90 Participants
are divided into 3 treatment groups

Sponsors and collaborators

Dyne Therapeutics

Lead sponsor

Source ClinicalTrials.gov