This study, called FORZETTO, is testing a drug called zeleciment rostudirsen (DYNE-251) for boys with Duchenne muscular dystrophy (DMD). It aims to see how well the drug works, if it's safe, and how well people tolerate it. Participants will receive either zeleciment rostudirsen or a placebo (an inactive substance) through an IV every four weeks. Boys between 4 and 18 years old who can walk and have a specific genetic mutation (exon 51 skipping) in their dystrophin gene may be able to join. The main way researchers will measure success is by looking at how quickly participants can rise from the floor. The study is currently unclear on its recruitment status and plans to enroll 90 participants.

Study design

This is an interventional study with a placebo-controlled period followed by an open-label long-term extension. It plans to enroll 90 male participants.

What's involved

Participants will have a screening period of up to 6 weeks, followed by a 72-week placebo-controlled period, and then a 96-week open-label extension period. Zeleciment rostudirsen or placebo will be administered by IV infusion every 4 weeks.

Compensation

Not stated in the trial record.

Follow-up

The primary endpoint, Rise From Floor (RFF) velocity, is measured at Baseline and Week 73.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.