protocolSection identificationModule nctId: NCT06270719, orgStudyIdInfo id: SRP-9001-401, briefTitle: An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy, acronym: ENDURE, statusModule overallStatus: RECRUITING, startDateStruct date: 2024-02-07, primaryCompletionDateStruct date: 2029-12-31, completionDateStruct date: 2038-12-31, studyFirstPostDateStruct date: 2024-02-21, lastUpdatePostDateStruct date: 2024-04-12, sponsorCollaboratorsModule leadSponsor name: Sarepta Therapeutics, Inc., class: INDUSTRY, descriptionModule briefSummary: 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. In addition, treatment outcomes will be collected prospectively from post-trial participants who have received delandistrogene moxeparvovec through participation in select SRP-9001 studies., conditionsModule conditions: Duchenne Muscular Dystrophy, designModule studyType: OBSERVATIONAL, designInfo observationalModel: COHORT, timePerspective: PROSPECTIVE, enrollmentInfo count: 500, type: ESTIMATED, armsInterventionsModule interventions name: delandistrogene moxeparvovec, interventions name: Standard of Care, outcomesModule primaryOutcomes measure: All Cohorts: Mean Change From Baseline in Time to Walk/Run 10 Meters (and Calculated Velocity) at Month 12, secondaryOutcomes measure: All Cohorts: Time to Rise From Floor (Supine to Stand), secondaryOutcomes measure: All Cohorts: Loss of Ambulation (LOA), secondaryOutcomes measure: All Cohorts: Performance of Upper Limb (PUL) Version 2.0 Entry Item Score, secondaryOutcomes measure: All Cohorts: Patient-reported Outcomes Measurement Information (PROMIS) Domain Scores of Mobility, Upper Extremity and Fatigue, secondaryOutcomes measure: All Cohorts: Pulmonary Function, as Measured Forced Vital Capacity (FVC) (% Predicted), secondaryOutcomes measure: All Cohorts: Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF) as Measured by Echocardiogram (ECHO), secondaryOutcomes measure: All Cohorts: Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs), secondaryOutcomes measure: Delandistrogene Moxeparvovec Cohorts: Time to Walk/Run 10 Meters (Calculated Velocity), secondaryOutcomes measure: Delandistrogene Moxeparvovec Post-trial Cohort: North Star Ambulatory Assessment (NSAA), eligibilityModule sex: MALE, minimumAge: 4 Years, stdAges: CHILD, stdAges: ADULT, stdAges: OLDER_ADULT, contactsLocationsModule locations facility: Arkansas Children's Hospital, status: RECRUITING, city: Little Rock, state: Arkansas, zip: 72202, country: United States, contacts role: CONTACT, email: SareptAlly@sarepta.com, contacts name: Aravindhan Veerapandiyan, MD, role: PRINCIPAL_INVESTIGATOR, geoPoint lat: 34.74648, lon: -92.28959, locations facility: Penn State Health Milton S. Hershey Medical Center, status: NOT_YET_RECRUITING, city: Hershey, state: Pennsylvania, zip: 17033, country: United States, contacts role: CONTACT, email: SareptAlly@sarepta.com, contacts name: Ashutosh Kumar, MD, role: PRINCIPAL_INVESTIGATOR, geoPoint lat: 40.28592, lon: -76.65025, locations facility: Cook Children's Hospital, status: RECRUITING, city: Fort Worth, state: Texas, zip: 76104, country: United States, contacts role: CONTACT, email: SareptAlly@sarepta.com, geoPoint lat: 32.72541, lon: -97.32085, locations facility: Children's Hospital of the King's Daughters, status: RECRUITING, city: Norfolk, state: Virginia, zip: 23507, country: United States, contacts role: CONTACT, email: SareptAlly@sarepta.com, contacts name: Crystal Proud, MD, role: PRINCIPAL_INVESTIGATOR, geoPoint lat: 36.84681, lon: -76.28522, hasResults: False