NCT lookup

Pull any trial record directly from ClinicalTrials.gov.

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

NCT07608432

RecruitingPhase 3

Sponsor: Dyne Therapeutics

Conditions: Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne, Muscular Dystrophy (DMD), DMD, Muscular Dystrophies

Interventions: Zeleciment Rostudirsen (DYNE-251), Placebo

Countries: United States

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 overview

Sex: MALE

Age: 4 Years to 18 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

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

Locations (1)

Hillsborough, North Carolina, United States