Zeleciment rostudirsen (DYNE-251) for boys and young people with Duchenne muscular dystrophy
Tests zeleciment rostudirsen in walking boys with Duchenne muscular dystrophy.
Plain-language title generated from the registry record
Registry title: Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
Key facts
- Where
- Hillsborough, United StatesSee the site
- Phase
- Phase 3
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 4 and 18 years old?
Source: NCT07608432, who can take part
Where in United States
- Rare Disease Research, LLCHillsborough, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Dyne Therapeutics
- Trial code
- NCT07608432EU 2025-524096-23-00
- Record updated
- 27 May 2026
- Original description
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.Read full original descriptionShow less
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
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.
Inclusion: 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: 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.