An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy (ENDURE)

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

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

Study Overview

• Status: Enrolling by invitation
• Conditions: Duchenne Muscular Dystrophy
• Intervention / Treatment: Genetic: Delandistrogene Moxeparvovec; Drug: Standard of Care

• Study Type: Observational
• Enrollment (Estimated): 500

Contacts and Locations

Study Locations

• United States
  • Arkansas
    • Little Rock, Arkansas, United States, 72202
      • Arkansas Children's Hospital
  • California
    • Los Angeles, California, United States, 90027
      • Children's Hospital Los Angeles - PIN
  • Colorado
    • Aurora, Colorado, United States, 80045
      • University of Colorado - PPDS
  • Connecticut
    • Hartford, Connecticut, United States, 06106
      • Connecticut Children's Medical Center - Hartford
  • District of Columbia
    • Washington D.C., District of Columbia, United States, 20010
      • Children's National Medical Center
  • Florida
    • Miami, Florida, United States, 33155
      • Nicklaus Children's Hospital
    • Orlando, Florida, United States, 32827
      • Nemours Children's Hospital - Orlando
    • St. Petersburg, Florida, United States, 33701
      • All Children's Research Institute, Inc
  • Illinois
    • Chicago, Illinois, United States, 60611-2991
      • Ann and Robert H Lurie Childrens Hospital of Chicago
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Indiana Clinical and Translational Science Institute
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • University of Kansas Medical Center
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan
  • North Carolina
    • Durham, North Carolina, United States, 27704
      • Duke Lenox Baker Children's Hospital
  • Ohio
    • Akron, Ohio, United States, 44302
      • Childrens Hospital Medical Center of Akron
  • Oregon
    • Portland, Oregon, United States, 97239
      • OHSU Healthcare (Oregon Health and Science University)
  • Pennsylvania
    • Hershey, Pennsylvania, United States, 17033
      • Penn State Health Milton S. Hershey Medical Center
    • Philadelphia, Pennsylvania, United States, 19104
      • Children's Hospital of Philadelphia
  • Tennessee
    • Memphis, Tennessee, United States, 38103
      • Le Bonheur Children's Hospital
    • Nashville, Tennessee, United States, 37232
      • Vanderbilt University Medical Center
  • Texas
    • Fort Worth, Texas, United States, 76104
      • Cook Children's Hospital
  • Virginia
    • Charlottesville, Virginia, United States, 22903
      • University of Virginia
    • Norfolk, Virginia, United States, 23507
      • Children's Hospital of the King's Daughters
  • Washington
    • Seattle, Washington, United States, 98105
      • Seattle Children's Hospital
  • Wisconsin
    • Madison, Wisconsin, United States, 53715-1218
      • The Board of Regents of the University of Wisconsin
    • Milwaukee, Wisconsin, United States, 53226-4874
      • The Medical College of Wisconsin

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

DMD participants within each prescriber's practice who either (1) have been prescribed delandistrogene moxeparvovec or (2) are receiving or have been prescribed to begin chronic glucocorticoid therapy in routine clinical practice, and who meet the study eligibility criteria, will be invited to enroll into the study and will be followed according to the protocol.

Description

Inclusion Criteria:

• Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test.
• Is currently receiving or has been prescribed to start chronic glucocorticoid therapy at the time of this observational study enrollment.

For ELEVIDYS-treated Participants (Cohorts 1a, 1b, and 1c):

• Is at least 4 years of age at the time of infusion
• Will either: a) be initiating or has initiated ELEVIDYS within the last 30 days in routine clinical practice at the time of this observational study enrollment, or b) was administered ELEVIDYS in routine clinical practice and has the required minimum dataset for entry into the observational study per Sponsor approval

For Standard of Care Comparators (Cohort 2):

• Is at least 4 years of age at the time of enrollment
• Is unexposed to DMD gene therapy at the time of this observational study enrollment

Exclusion Criteria:

• Has any deletion of exon 8 and/or exon 9 in the DMD gene.
• Is currently participating in any DMD interventional study at the time of this observational study enrollment.
• Has any prior exposure to DMD gene therapy other than that described for Cohort 1c (ELEVIDYS Retrospectively Treated Cohort).

• Has a medical condition or confounding circumstances (for example, prior traumatic limitation for mobility or significant behavioral comorbidity) that, in the opinion of the Investigator, might compromise:

  • The participant's ability to comply with the protocol-required procedures,
  • The participant's wellbeing or safety, and/or
  • The clinical interpretability of the data collected from the participant.

Other inclusion/exclusion criteria may apply.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Cohort 1 (Treated); Cohort 1: participants prescribed ELEVIDYS in a commercial setting. Cohort 1a (Ambulatory ELEVIDYS Prospectively Treated Cohort): participants prescribed ELEVIDYS by commercially treating physicians with consent no later than 30 days from infusion. All participants will be dosed with ELEVIDYS based on United States prescribing information (USPI). The Post Market Requirement (PMR) Cohort (sub-cohort of 1a) consists of participants with laboratory data as specified in the ELEVIDYS USPI. Cohort 1b (Non-ambulatory ELEVIDYS Prospectively Treated Cohort): non-ambulatory DMD participants prescribed ELEVIDYS commercially and recruited by treating physicians before infusion (enrollment currently closed). Cohort 1c (ELEVIDYS Retrospectively Treated Cohort): participants dosed with ELEVIDYS with complete baseline data within 6 months prior to dosing and complete prospectively collected annual follow-up data after infusion until the time of cohort entry (sponsor approval required for enrollment).	Genetic: Delandistrogene Moxeparvovec; No study medication will be provided by the sponsor during this study.; Other Names: SRP-9001; delandistrogene moxeparvovec-rokl; ELEVIDYS
Cohort 2 (Standard of Care); Cohort 2: ambulatory DMD participants who are at least 4 years of age at baseline, unexposed to DMD gene therapy, and receiving or prescribed chronic glucocorticoids at study entry.	Drug: Standard of Care; No study medication will be provided by the sponsor during this study.; Other Names: Chronic glucocorticoid treatment

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Mean Change From Baseline in Time to Walk/Run 10 Meters (10MWR) (Calculated Velocity) at Month 12	Baseline, Month 12
Number of Participants Experiencing Acute Liver Injury (ALI)	Baseline through Month 12

Secondary Outcome Measures

Outcome Measure	Time Frame
Time to Rise From Floor (Supine to Stand)	Up to 10 years
Loss of Ambulation (LOA)	Up to 10 years
Performance of Upper Limb (PUL) Version 2.0 Entry Item A Score or Brooke Upper Extremity Scale Score	Up to 10 years
Patient-reported Outcomes Measurement Information (PROMIS) Domain Scores of Mobility, Upper Extremity, and Fatigue	Up to 10 years
Pulmonary Function as Measured by Forced Vital Capacity (FVC)	Up to 10 years
Pulmonary Function as Measured by FVC Percent Predicted (FVC%p)	Up to 10 years
Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF), as Measured by Echocardiogram (ECHO) or Cardiac MRI (cMRI)	Up to 10 years
Number of Participants Experiencing Serious ALI and Acute Liver Failure (ALF)	Baseline through Month 12
Number of Participants Experiencing Complications Associated with ALI and ALF	Up to 10 years
Survival Time	Up to 10 years
Time to First Vertebral Body (Spine) Fracture	Up to 10 years

Collaborators and Investigators

• Sponsor: Sarepta Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): February 7, 2024
• Primary Completion (Estimated): December 31, 2029
• Study Completion (Estimated): December 31, 2038

Study Registration Dates

• First Submitted: January 24, 2024
• First Submitted That Met QC Criteria: February 13, 2024
• First Posted (Actual): February 21, 2024

Study Record Updates

• Last Update Posted (Actual): May 4, 2026
• Last Update Submitted That Met QC Criteria: April 28, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: Observational; Duchenne Muscular Dystrophy; Standard of Care; DMD; Phase 4; ELEVIDYS
• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Muscular Disorders, Atrophic; Muscular Dystrophies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Muscular Dystrophy, Duchenne; Health Services Administration; Health Care Quality, Access, and Evaluation; Quality of Health Care; Quality Indicators, Health Care; Standard of Care
• Other Study ID Numbers: SRP-9001-401

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No