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