Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
Recruiting
Phase 3Interventional Study
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)
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
4 – 18
Sex
Male
Trial phase
Phase 3
Study type
Interventional
Purpose
Treatment
Participants needed
90 (estimated)
Sponsor
Dyne Therapeutics · Industry
Who this trial is looking for
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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.
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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 do…
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
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