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NCT06270719

A Long-term Multicenter Prospective Observational Study Evaluating the Comparative Effectiveness and Safety of Sarepta Gene Transfer Therapy vs. Standard of Care in Participants With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice

Study Summary

This is a multicenter, prospective, observational Phase 4 study in the United States. The study is designed to collect both medical history and prospective data on Duchenne muscular dystrophy (DMD) treatment outcomes in participants receiving delandistrogene moxeparvovec as part of clinical care, compared to participants with DMD receiving or prescribed to start chronic glucocorticoid treatment at baseline in routine clinical practice.

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Interventions

Delandistrogene MoxeparvovecGENETIC
No study medication will be provided by the sponsor during this study.
Standard of CareDRUG
No study medication will be provided by the sponsor during this study.

Study Locations

FacilityCityStateCountry
Arkansas Children's HospitalLittle RockArkansasUnited States
Children's Hospital Los Angeles - PINLos AngelesCaliforniaUnited States
University of Colorado - PPDSAuroraColoradoUnited States
Connecticut Children's Medical Center - HartfordHartfordConnecticutUnited States
Children's National Medical CenterWashington D.C.District of ColumbiaUnited States
Nicklaus Children's HospitalMiamiFloridaUnited States
Nemours Children's Hospital - OrlandoOrlandoFloridaUnited States
All Children's Research Institute, IncSt. PetersburgFloridaUnited States
Ann and Robert H Lurie Childrens Hospital of ChicagoChicagoIllinoisUnited States
Indiana Clinical and Translational Science InstituteIndianapolisIndianaUnited States
University of Kansas Medical CenterKansas CityKansasUnited States
University of MichiganAnn ArborMichiganUnited States
Duke Lenox Baker Children's HospitalDurhamNorth CarolinaUnited States
Childrens Hospital Medical Center of AkronAkronOhioUnited States
OHSU Healthcare (Oregon Health and Science University)PortlandOregonUnited States
Penn State Health Milton S. Hershey Medical CenterHersheyPennsylvaniaUnited States
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvaniaUnited States
Le Bonheur Children's HospitalMemphisTennesseeUnited States
Vanderbilt University Medical CenterNashvilleTennesseeUnited States
Cook Children's HospitalFort WorthTexasUnited States
University of VirginiaCharlottesvilleVirginiaUnited States
Children's Hospital of the King's DaughtersNorfolkVirginiaUnited States
Seattle Children's HospitalSeattleWashingtonUnited States
The Board of Regents of the University of WisconsinMadisonWisconsinUnited States
The Medical College of WisconsinMilwaukeeWisconsinUnited States

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026