NCT04626674

An Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects With Duchenne Muscular Dystrophy (ENDEAVOR)

Study Summary

Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed. This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with Duchenne Muscular Dystrophy (DMD). The maximum participant duration for this study is 156 weeks.

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Interventions

delandistrogene moxeparvovecGENETIC
Single IV infusion of delandistrogene moxeparvovec

Study Locations

FacilityCityStateCountry
Arkansas Children's HospitalLittle RockArkansasUnited States
University of California, San DiegoLa JollaCaliforniaUnited States
University of California, Los AngelesLos AngelesCaliforniaUnited States
Stanford UniversityPalo AltoCaliforniaUnited States
University of California, DavisSacramentoCaliforniaUnited States
University of FloridaGainesvilleFloridaUnited States
Ann & Robert H. Lurie Children's Hospital of ChicagoChicagoIllinoisUnited States
Washington University in St. LouisSt LouisMissouriUnited States
Duke University Medical CenterDurhamNorth CarolinaUnited States
Nationwide Children's HospitalColumbusOhioUnited States
Neurology Rare Disease CenterFlower MoundTexasUnited States
Children's Hospital of The King's DaughtersNorfolkVirginiaUnited States

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: October 12, 2026