Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Zeleciment Rostudirsen (DYNE-251), Placebo.
- Who it may be relevant to
- Registry conditions: Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne, Muscular Dystrophy (DMD), DMD. Basic parameters: 4 years — 18 years · Male.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of DYNE-251 Administered Intravenously in Ambulatory Male Participants 4 to 18 Years of Age With Duchenne Muscular Dystrophy Amenable to Exon-51 Skipping
Overview
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.
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).
Interventions
- Drug Zeleciment Rostudirsen (DYNE-251)
Administered by IV infusion - Drug Placebo
Administered by IV infusion
Primary outcome measures
- Rise From Floor (RFF) velocity [Time frame: Baseline, Week 73]
Secondary outcome measures (12)
- RFF (Rise From Floor) velocity [Time frame: Baseline, up to Week 169]
- Stride Velocity 95th Percentile (SV95C) [Time frame: Baseline, Week 73, up to Week 169]
- North Star Ambulatory Assessment (NSAA) Total Score [Time frame: Baseline, Week 73, up to Week 169]
- 10-Meter Walk/Run (10MWR) Velocity [Time frame: Baseline, Week 73, up to Week 169]
- 4-Stair Climb (4SC) velocity [Time frame: Baseline, Week 73, up to Week 169]
- Functional Composite score [Time frame: Baseline, Week 73, up to Week 169]
- Forced Vital Capacity (FVC) [Time frame: Baseline, Week 73, up to Week 169]
- Patient Global Impression of Severity (PGI-S) [Time frame: Baseline, Week 73, up to Week 169]
- Outcome of Patient Global Impression of Change (PGI-C) [Time frame: Week 73, up to Week 169]
- Blood Creatine Kinase (CK) levels [Time frame: Baseline, Week 73, up to Week 169]
- Incidence of participants With Treatment-Emergent Adverse Events (TEAEs) [Time frame: Through study completion, up to Week 173]
- Maximum Observed Plasma Drug Concentration of DYNE-251 (Cmax) [Time frame: Through study completion, up to Week 169]
Eligibility 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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
United States · 1 center
- Rare Disease Research, LLC — Hillsborough
Identifiers
NCT: NCT07608432 · DYNE251-DMD-301 · 2025-524096-23-00