Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
Purpose
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
- 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
- Eligible Ages
- Between 4 Years and 18 Years
- Eligible Sex
- Male
- Accepts Healthy Volunteers
- No
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
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
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. |
|
Recruiting Locations
Rare Disease Research, LLC
Hillsborough, North Carolina 27278
Hillsborough, North Carolina 27278
More Details
- Status
- Recruiting
- Sponsor
- Dyne Therapeutics
Detailed Description
The study consists of three periods: a Screening period (up to 6 weeks), a Placebo-Controlled Period (72 weeks) and an open-label Long-Term Extension Period (96 weeks).