Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)

Part of paid clinical trials in Hillsborough, North Carolina.

Sponsor
Dyne Therapeutics
Study ID
NCT07608432
Phase
PHASE3
Status
Recruiting
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Conditions

  • Congenital, Hereditary, and Neonatal Diseases and Abnormalities
  • DMD
  • Duchenne Muscular Dystrophy (DMD)
  • Genetic Disease, Inborn
  • Genetic Disease, X-Linked
  • Muscular Dystrophies
  • Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)
  • Muscular Dystrophy (DMD)
  • Muscular Dystrophy in Children
  • Muscular Dystrophy, Duchenne
  • Muscular Dystrophy, Duchenne Type
  • Muscular Dystrophy, Duchenne and Becker Types
  • Neuromuscular Diseases (NMD)

Eligibility Criteria

Sex
MALE
Age
4 Years - 18 Years
Healthy Volunteers
Not accepted

Interventions

  • Zeleciment Rostudirsen (DYNE-251) — DRUG
    Administered by IV infusion
  • Placebo — DRUG
    Administered by IV infusion

Study Details

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.

Key Dates

Start date
Jun 30, 2026
Status verified
May 2026
Primary completion
Dec 31, 2030
Completion
Oct 31, 2032

Study Design

Enrollment
90 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

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.

Primary Outcome Measure

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

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Rare Disease Research, LLCHillsboroughNorth Carolina27278
Hannah Nation
[email protected]
984-314-2252

Find similar trials in Hillsborough, NC

By condition
Duchenne muscular dystrophy
By specialty
Pediatrics
By research site
Rare Disease Research, LLC· Hillsborough, NC

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