Zeleciment Rostudirsen (DYNE-251) for Duchenne Muscular Dystrophy in Ambulatory Boys

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 3, randomized study evaluates the efficacy, safety, and tolerability of DYNE-251 given IV every 4 weeks in ambulatory male participants aged 4 to 18 with Duchenne muscular dystrophy due to dystrophin mutations amenable to exon 51 skipping. The study has three parts: screening, a 72-week placebo-controlled period, and a 96-week open-label extension to assess long-term outcomes. Participants will continue stable glucocorticoid therapy during the placebo-controlled period unless weight changes require adjustments. Key eligibility includes ambulatory status, exon 51 skipping amenable mutation, and adequate reach-to-rise performance; exclusion criteria rule out recent immunosuppressants and other muscle-active therapies.

Why it matters

Who can join: the full rules

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

Where it's running

• Rare Disease Research, LLC
  Hillsborough, North Carolina, United States Recruiting

Study contacts

• Study coordinator: Dyne Clinical Trials
• Email: clinicaltrials@dyne-tx.com
• Phone: +1-781-317-1919

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.