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Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
Sponsor: Dyne Therapeutics
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.
Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of DYNE-251 Administered Intravenously in Ambulatory Male Participants 4 to 18 Years of Age With Duchenne Muscular Dystrophy Amenable to Exon-51 Skipping
Key Details
Gender
MALE
Age Range
4 Years - 18 Years
Study Type
INTERVENTIONAL
Enrollment
90
Start Date
2026-06
Completion Date
2032-10
Last Updated
2026-05-27
Healthy Volunteers
No
Conditions
Interventions
Zeleciment Rostudirsen (DYNE-251)
Administered by IV infusion
Placebo
Administered by IV infusion
Locations (1)
Rare Disease Research, LLC
Hillsborough, North Carolina, United States