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

Recruiting Phase 3 Interventional Study
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)
Pivotal Trial This treatment is in the last trial phase before FDA approval. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
Ready to participate?

Review the details below, then apply to join this clinical trial.

At a Glance
Age
4 – 18
Sex
Male
Trial phase
Phase 3
Study type
Interventional
Purpose
Treatment
Participants needed
90 (estimated)
Sponsor
Dyne Therapeutics · Industry
Think this trial could be right for you?

Answer a few quick questions to see if you may meet the eligibility requirements.

Check Your Eligibility
About This Trial

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.

Trial Locations
Loading…

Loading trial locations…

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 do…
Contacts

Dyne Clinical Trials

+1-781-317-1919

clinicaltrials@dyne-tx.com

CONTACT