Gene therapy vs. standard care: new study tracks Long-Term outcomes in duchenne patients

NCT ID NCT06270719

ENROLLING_BY_INVITATION Disease control Sponsor: Sarepta Therapeutics, Inc. Source: ClinicalTrials.gov ↗

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

Summary

This study follows 500 people with Duchenne muscular dystrophy to see how well a gene therapy (ELEVIDYS) works compared to standard steroid treatment over time. Researchers will measure movement abilities, breathing, and safety, including liver problems. Participants must already be on or starting steroids, and those getting gene therapy must be at least 4 years old.

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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

All Children's Research Institute, Inc

St. Petersburg, Florida, 33701, United States
Ann and Robert H Lurie Childrens Hospital of Chicago

Chicago, Illinois, 60611-2991, United States
Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States
Children's Hospital Los Angeles - PIN

Los Angeles, California, 90027, United States
Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of the King's Daughters

Norfolk, Virginia, 23507, United States
Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States
Childrens Hospital Medical Center of Akron

Akron, Ohio, 44302, United States
Connecticut Children's Medical Center - Hartford

Hartford, Connecticut, 06106, United States
Cook Children's Hospital

Fort Worth, Texas, 76104, United States
Duke Lenox Baker Children's Hospital

Durham, North Carolina, 27704, United States
Indiana Clinical and Translational Science Institute

Indianapolis, Indiana, 46202, United States
Le Bonheur Children's Hospital

Memphis, Tennessee, 38103, United States
Nemours Children's Hospital - Orlando

Orlando, Florida, 32827, United States
Nicklaus Children's Hospital

Miami, Florida, 33155, United States
OHSU Healthcare (Oregon Health and Science University)

Portland, Oregon, 97239, United States
Penn State Health Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States
Seattle Children's Hospital

Seattle, Washington, 98105, United States
The Board of Regents of the University of Wisconsin

Madison, Wisconsin, 53715-1218, United States
The Medical College of Wisconsin

Milwaukee, Wisconsin, 53226-4874, United States
University of Colorado - PPDS

Aurora, Colorado, 80045, United States
University of Kansas Medical Center

Kansas City, Kansas, 66160, United States
University of Michigan

Ann Arbor, Michigan, 48109, United States
University of Virginia

Charlottesville, Virginia, 22903, United States
Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

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