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RECRUITING
NCT07608432
PHASE3

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

Sponsor: Dyne Therapeutics

View on ClinicalTrials.gov

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.

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

Key Details

Gender

MALE

Age Range

4 Years - 18 Years

Study Type

INTERVENTIONAL

Enrollment

90

Start Date

2026-06

Completion Date

2032-10

Last Updated

2026-05-27

Healthy Volunteers

No

Interventions

DRUG

Zeleciment Rostudirsen (DYNE-251)

Administered by IV infusion

DRUG

Placebo

Administered by IV infusion

Locations (1)

Rare Disease Research, LLC

Hillsborough, North Carolina, United States