New hope for duchenne kids: drug combo after gene therapy tested

NCT ID NCT06100887

Ongoing Disease control Sponsor: Edgewise Therapeutics, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests an experimental drug called EDG-5506 (sevasemten) in 43 children and teens aged 6 to 17 with Duchenne muscular dystrophy who have already received gene therapy. The goal is to see if the drug is safe and how it affects muscle damage markers. Participants are randomly assigned to receive the drug or a placebo, and later everyone can receive the drug.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for DUCHENNE MUSCULAR DYSTROPHY are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DUCHENNE MUSCULAR DYSTROPHY

Contacts and locations

Locations

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States
Rare Disease Research

Hillsborough, North Carolina, 27278, United States
UC Davis Medical Center

Sacramento, California, 95817, United States
UCLA Medical Center

Los Angeles, California, 90095, United States
University of Florida

Gainesville, Florida, 32610, United States
University of Massachusetts Memorial Medical Center

Worcester, Massachusetts, 01605, United States
Washington University School of Medicine

St Louis, Missouri, 63110, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.