Dyne Therapeutics has announced the start of the Dyne Therapeutics Phase 3 FORZETTO Trial (NCT07608432) of Z-Rostudirsen for DMD, marking another major step forward for families affected by Duchenne muscular dystrophy (DMD).
The global Phase 3 study will evaluate z-rostudirsen (DYNE-251) in people living with DMD who are amenable to exon 51 skipping. The company also plans to submit a Biologics License Application (BLA) to the U.S. FDA later this quarter for accelerated approval. Learn More: What does BLA mean?
The FORZETTO trial design will be presented during the 19th International Congress on Neuromuscular Diseases (ICNMD) in Florence, Italy.
Table of Contents
What Is Z-Rostudirsen (DYNE-251)?
A Next-Generation Exon 51 Skipping Therapy
Z-rostudirsen, also known as DYNE-251, is an investigational therapy designed for people with DMD mutations that can benefit from exon 51 skipping.
The therapy aims to help the body produce near-full-length dystrophin protein across muscle tissues and the central nervous system. Dystrophin is essential for protecting muscles from damage, and its absence leads to progressive muscle weakness in DMD patients.
According to Dyne Therapeutics, the goal of the treatment is not only to improve laboratory biomarkers, but also to help patients move better, breathe better, and improve daily quality of life.
TRY NOW: DMD or BMD? Exon Check Tool
How the FORZETTO Phase 3 Trial Will Work
Global Randomized Placebo-Controlled Study
The DYNE-251 Phase 3 FORZETTO Trial is a global, randomized, double-blind, placebo-controlled study designed to evaluate the safety and effectiveness of z-rostudirsen.
Key details of the study include:
- Approximately 90 ambulatory male participants
- Ages 4 to 18 years
- Participants must be amenable to exon 51 skipping
- Patients will receive either:
- 20 mg/kg z-rostudirsen
- or placebo
- Treatment will be given every four weeks (Q4W)
The first clinical trial site has already been activated and enrollment is now open.
Primary Endpoint Focuses on Muscle Function
Rise From Floor Velocity Will Be Measured
The main goal of the study is to measure changes in rise from floor (RFF) velocity, also called time to rise (TTR) velocity, at Week 73.
This test measures how quickly a patient can stand up from the floor and is considered an important indicator of muscle strength and motor coordination in DMD clinical trials.
In the earlier Phase 1/2 DELIVER study, patients treated with z-rostudirsen showed meaningful improvements in RFF velocity after six months compared with placebo-treated participants.
Additional Secondary Endpoints in the FORZETTO Study
Researchers Will Track Mobility and Lung Function
The study will also evaluate several additional functional outcomes, including:
- Stride Velocity 95th Centile (SV95C)
- North Star Ambulatory Assessment (NSAA)
- 10-Meter Walk/Run (10MWR)
- Four-Stair Climb (4SC)
- Forced Vital Capacity Percent Predicted (FVC%p)
Researchers will also collect patient-reported outcomes to better understand how treatment may impact daily life.
After the initial 72-week placebo-controlled period, eligible participants may continue into a 96-week open-label extension study.
FDA Alignment and Accelerated Approval Plans
FORZETTO Could Support Full Traditional Approval
Dyne Therapeutics stated that the company has aligned with the U.S. Food and Drug Administration (FDA) on the FORZETTO Phase 3 trial design and protocol.
The study is intended to serve as the confirmatory trial required to potentially convert accelerated approval into full traditional approval in the United States. The trial may also support future regulatory applications outside the U.S.
Why the Dyne Therapeutics Phase 3 FORZETTO Trial of Z-Rostudirsen for DMD Matters
A Potentially Important Development for the DMD Community
The launch of the Dyne Therapeutics Phase 3 FORZETTO Trial of Z-Rostudirsen for DMD represents an important milestone for the Duchenne community, especially for patients eligible for exon 51 skipping therapies.
With promising earlier clinical data and a large confirmatory study now underway, researchers and families will be closely watching whether DYNE-251 can deliver meaningful functional improvements for people living with Duchenne muscular dystrophy.
Contact Information
- Name: Dyne Clinical Trials
- Phone Number: +1-781-317-1919
- Email: [email protected]
Follow This Page >>> All Clinical Trials for Duchenne
