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

April 11, 2024 updated by: Sarepta Therapeutics, Inc.

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 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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Estimated)

500

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4
  • Phone Number: 1-888-SAREPTA (1-888-727-3782)
  • Email: SareptAlly@sarepta.com

Study Locations

  • United States
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Recruiting
        • Arkansas Children's Hospital
        • Principal Investigator:
          • Aravindhan Veerapandiyan, MD
        • Contact:
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Not yet recruiting
        • Penn State Health Milton S. Hershey Medical Center
        • Contact:
        • Principal Investigator:
          • Ashutosh Kumar, MD
    • Texas
      • Fort Worth, Texas, United States, 76104
    • Virginia
      • Norfolk, Virginia, United States, 23507
        • Recruiting
        • Children's Hospital of The King's Daughters
        • Principal Investigator:
          • Crystal Proud, MD
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

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 a definitive diagnosis of DMD prior to Screening based on documentation of clinical findings and confirmatory genetic testing.

Among participants recruited from routine clinical practice:

  • Is aged 4 through 5 years at the time of enrollment.
  • Is ambulatory per protocol specified criteria.
  • Is currently receiving or has been prescribed to start chronic glucocorticoid therapy at the time of this observational study enrollment.

For Delandistrogene Moxeparvovec-exposed Participants:

- Will be initiating usual care treatment with delandistrogene moxeparvovec at the time of study enrollment.

For Comparators:

- Is unexposed to DMD gene therapy at the time of study enrollment.

Exclusion Criteria:

Among participants recruited from routine clinical practice:

  • 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 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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Delandistrogene Moxeparvovec
Participants were either (1) prescribed delandistrogene moxeparvovec commercially as part of clinical care prior to entry into this trial or (2) previously received delandistrogene moxeparvovec through participation in select SRP-9001 studies.
Genetic: delandistrogene moxeparvovec
No study medication will be provided by the sponsor during this trial.
Other Names:
  • SRP-9001
  • delandistrogene moxeparvovec-rokl
  • ELEVIDYS
Standard of Care
Participants unexposed to DMD gene therapy receiving standard of care therapy (chronic glucocorticoid treatment) that does not include delandistrogene moxeparvovec or other DMD gene therapies.
Drug: Standard of Care
No study medication will be provided by the sponsor during this trial.
Other Names:
  • Chronic glucocorticoid treatment

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
All Cohorts: Mean Change From Baseline in Time to Walk/Run 10 Meters (and Calculated Velocity) at Month 12
Time Frame: Baseline, Month 12
Baseline, Month 12

Secondary Outcome Measures

Outcome Measure
Time Frame
All Cohorts: Time to Rise From Floor (Supine to Stand)
Time Frame: Up to 10 years
Up to 10 years
All Cohorts: Loss of Ambulation (LOA)
Time Frame: Up to 10 years
Up to 10 years
All Cohorts: Performance of Upper Limb (PUL) Version 2.0 Entry Item Score
Time Frame: Up to 10 years
Up to 10 years
All Cohorts: Patient-reported Outcomes Measurement Information (PROMIS) Domain Scores of Mobility, Upper Extremity and Fatigue
Time Frame: Up to 10 years
Up to 10 years
All Cohorts: Pulmonary Function, as Measured Forced Vital Capacity (FVC) (% Predicted)
Time Frame: Up to 10 years
Up to 10 years
All Cohorts: Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF) as Measured by Echocardiogram (ECHO)
Time Frame: Up to 10 years
Up to 10 years
All Cohorts: Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs)
Time Frame: Up to 10 years
Up to 10 years
Delandistrogene Moxeparvovec Cohorts: Time to Walk/Run 10 Meters (Calculated Velocity)
Time Frame: Up to 10 years
Up to 10 years
Delandistrogene Moxeparvovec Post-trial Cohort: North Star Ambulatory Assessment (NSAA)
Time Frame: Up to 10 years
Up to 10 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sarepta Therapeutics, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

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)

April 12, 2024

Last Update Submitted That Met QC Criteria

April 11, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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