Recruiting
Phase 3

Zeleciment Rostudirsen

Sponsor:

Dyne Therapeutics

Code:

NCT07608432

Conditions

Duchenne Muscular Dystrophy (DMD)

Muscular Dystrophy, Duchenne

Muscular Dystrophy (DMD)

DMD

Muscular Dystrophies

Eligibility Criteria

Sex: Male

Age: 4 - 18

Healthy Volunteers: Not accepted

Interventions

Zeleciment Rostudirsen (DYNE-251)

Placebo

Study Details

Brief 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.

Conditions

Duchenne Muscular Dystrophy (DMD)

Muscular Dystrophy, Duchenne

Muscular Dystrophy (DMD)

DMD

Muscular Dystrophies

Study ID

NCT07608432

Start date

Jun, 2026

Status verified date

May, 2026

Completion date

Oct, 2032

Anticipated

Primary completion date

Dec, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Male

Age: 4 - 18

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

90 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Placebo-Controlled Period: Zeleciment Rostudirsen (DYNE-251)

Participants will be randomized to receive zeleciment rostudirsen, once every 4 weeks (Q4W) for up to 72 weeks.

placebo comparator: Placebo-Controlled Period: Placebo

Participants will be randomized to receive placebo, Q4W for up to 72 weeks.

experimental: Open-Label Long-Term Extension Period: Zeleciment Rostudirsen (DYNE-251)

All participants who complete the Placebo-Controlled Period of the study will receive zeleciment rostudirsen administered Q4W for up to 96 weeks.

Interventions

Zeleciment Rostudirsen (DYNE-251)

Administered by IV infusion

Placebo

Administered by IV infusion

Primary outcome measure

  • Rise From Floor (RFF) velocity [ Time Frame: Baseline, Week 73 ]

Central Contacts and Locations

Central contacts

Dyne Clinical Trials

+1-781-317-1919clinicaltrials@dyne-tx.com

Locations

Rare Disease Research, LLC

Recruiting

Hillsborough, North Carolina, United States, 27278

Contacts

Hannah Nation

984-314-2252hannah.nation@rarediseaseresearch.com

More Information

Sponsor

Dyne Therapeutics

Last update posted

May 27, 2026

Last verified

May, 2026

Keywords

  • Ambulatory
  • DMD
  • Duchenne Muscular Dystrophy
  • Duchenne
  • Dyne
  • Dyne Therapeutics
  • DYNE-251
  • Dystrophy
  • Exon Skipping
  • Exon 51
  • FORZETTO
  • Pediatric
  • PMO
  • Muscle Function
  • Muscular Dystropy, Duchenne
  • Rise From Floor
  • RFF
  • RFF Velocity
  • Rostudirsen
  • Time to rise
  • TTR
  • TTR Velocity
  • Zeleciment rostudirsen
  • Z-rostudirsen

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Dyne Therapeutics on 2026-05-27.