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Enrolling by Invitation

NCT Number: NCT06270719

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

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.

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Key information

Age range

4 year and older

Sex eligibility

Male

Study type

Observational

Primary location

Arkansas Children's Hospital, Little Rock, Arkansas, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

delandistrogene moxeparvovec

Genetic

No study medication will be provided by the sponsor during this study.

Other names: SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS

Standard of care

Drug

No study medication will be provided by the sponsor during this study.

Other names: Chronic glucocorticoid treatment

Primary outcomes

  1. Mean Change From Baseline in Time to Walk/Run 10 Meters (10MWR) (Calculated Velocity) at Month 12

    Time frame: Baseline, Month 12

  2. Number of Participants Experiencing Acute Liver Injury (ALI)

    Time frame: Baseline through Month 12

Secondary outcomes

  1. Time to Rise From Floor (Supine to Stand)

    Time frame: Up to 10 years

  2. Loss of Ambulation (LOA)

    Time frame: Up to 10 years

  3. Performance of Upper Limb (PUL) Version 2.0 Entry Item A Score or Brooke Upper Extremity Scale Score

    Time frame: Up to 10 years

  4. Patient-reported Outcomes Measurement Information (PROMIS) Domain Scores of Mobility, Upper Extremity, and Fatigue

    Time frame: Up to 10 years

  5. Pulmonary Function as Measured by Forced Vital Capacity (FVC)

    Time frame: Up to 10 years

  6. Pulmonary Function as Measured by FVC Percent Predicted (FVC%p)

    Time frame: Up to 10 years

  7. Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF), as Measured by Echocardiogram (ECHO) or Cardiac MRI (cMRI)

    Time frame: Up to 10 years

  8. Number of Participants Experiencing Serious ALI and Acute Liver Failure (ALF)

    Time frame: Baseline through Month 12

  9. Number of Participants Experiencing Complications Associated with ALI and ALF

    Time frame: Up to 10 years

  10. Survival Time

    Time frame: Up to 10 years

  11. Time to First Vertebral Body (Spine) Fracture

    Time frame: Up to 10 years

Sponsors and collaborators

Lead sponsor

Sarepta Therapeutics, Inc.

Industry

Registry information

Official study title

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

Acronym: ENDURE

Important dates

Study start
2024
Primary completion
2029
Study completion
2038
First posted
Feb 21, 2024
Registry last updated
May 4, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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