Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
Dyne Therapeutics · NCT07608432
Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.
Key facts
- Compensation
- Not posted. Ask the study team before screening.
- Who can join
- Ages 4–18 · Men only
- Healthy volunteers
- Not accepted (a condition is required)
- Study type
- Interventional · Phase 3
- Target enrollment
- 90
- US sites recruiting
- 1
- Study start
- June 2026
- Record last updated
- May 27, 2026
Status re-checked against ClinicalTrials.gov within the last 12 hours.
What the study is about
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.
Being studied: Zeleciment Rostudirsen (DYNE-251); Placebo
Conditions: Duchenne Muscular Dystrophy (DMD); Muscular Dystrophy, Duchenne; Muscular Dystrophy (DMD); DMD; Muscular Dystrophies; Muscular Dystrophy in Children; Muscular Dystrophy, Duchenne Type; Muscular Dystrophy, Duchenne and Becker Types; Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy); Genetic Disease, Inborn; Genetic Disease, X-Linked; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Neuromuscular Diseases (NMD)
Eligibility criteria
Copied from the official record. The study team makes the final decision at screening.
Show full 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
Recruiting US locations
Rare Disease Research, LLC
Hillsborough, NC 27278
Site contact: Hannah Nation · 984-314-2252 · hannah.nation@rarediseaseresearch.com
Study contacts (from the official record)
- Dyne Clinical Trials · +1-781-317-1919 · clinicaltrials@dyne-tx.com