Duchenne Muscular DystrophyENDURENCT06270719
An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy
An observational study of Duchenne Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 29 sites in United States.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 4 years and older, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Delandistrogene Moxeparvovec
- Type of treatment
- Injection or infusion
- How many people
- 500 participants
- Where
- 29 sitesUnited States
- Study dates
- 7 Feb 2024to 31 Dec 2038
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
29 sites in 1 country. Contact the study team or a site near you to ask about taking part.
29 sites in 1 country
United States29 sites
Childrens Hospital Medical Center of Akron
Akron, Ohio, 44308
UNM Clinical and Translational Science Center
Albuquerque, New Mexico, 87131-0001
University of Michigan
Ann Arbor, Michigan, 48109
University of Colorado - PPDS
Aurora, Colorado, 80045
Children's of Alabama
Birmingham, Alabama, 35233-1701
Boston Children's Hospital
Boston, Massachusetts, 02115
University of Virginia
Charlottesville, Virginia, 22903
Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, Illinois, 60611-2991
Duke Lenox Baker Children's Hospital
Durham, North Carolina, 27705-4504
Cook Children's Hospital
Fort Worth, Texas, 76104
Connecticut Children's Medical Center - Hartford
Hartford, Connecticut, 06106-3322
Penn State Health Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
Indiana Clinical and Translational Science Institute
Indianapolis, Indiana, 46202
University of Kansas Medical Center
Kansas City, Kansas, 66160
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Children's Hospital Los Angeles
Los Angeles, California, 90027
Norton Children's Neuroscience Institute - Louisville
Louisville, Kentucky, 40202
The Board of Regents of the University of Wisconsin
Madison, Wisconsin, 53715-1218
Nicklaus Children's Hospital
Miami, Florida, 33155
The Medical College of Wisconsin
Milwaukee, Wisconsin, 53226-4874
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23507
Nemours Children's Hospital - Orlando
Orlando, Florida, 32827
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
OHSU Healthcare (Oregon Health and Science University)
Portland, Oregon, 97239
Seattle Children's Hospital
Seattle, Washington, 98105
All Children's Research Institute, Inc
St. Petersburg, Florida, 33701
Stony Brook Medicine Advanced Specialty Care
Stony Brook, New York, 11794-3369
Children's National
Washington D.C., District of Columbia, 20010
About this study
Official 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
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.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Delandistrogene Moxeparvovec, Standard of Care
- Keywords
- Duchenne Muscular Dystrophy, DMD, Observational, Standard of Care, Phase 4, ELEVIDYS
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Delandistrogene MoxeparvovecGenetic
Also called SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS
No study medication will be provided by the sponsor during this study.
Standard of CareDrug
Also called Chronic glucocorticoid treatment
No study medication will be provided by the sponsor during this study.
| Study group | Role | What they receive |
|---|---|---|
| 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). Receives: Genetic: Delandistrogene Moxeparvovec | |
| 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. Receives: Drug: Standard of Care |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean Change From Baseline in Time to Walk/Run 10 Meters (10MWR) (Calculated Velocity) at Month 12
Measured: Baseline, Month 12
Number of Participants Experiencing Acute Liver Injury (ALI)
Measured: Baseline through Month 12
Secondary outcomes (11)
Time to Rise From Floor (Supine to Stand)
Measured: Up to 10 years
Loss of Ambulation (LOA)
Measured: Up to 10 years
Performance of Upper Limb (PUL) Version 2.0 Entry Item A Score or Brooke Upper Extremity Scale Score
Measured: Up to 10 years
Patient-reported Outcomes Measurement Information (PROMIS) Domain Scores of Mobility, Upper Extremity, and Fatigue
Measured: Up to 10 years
Pulmonary Function as Measured by Forced Vital Capacity (FVC)
Measured: Up to 10 years
Pulmonary Function as Measured by FVC Percent Predicted (FVC%p)
Measured: Up to 10 years
Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF), as Measured by Echocardiogram (ECHO) or Cardiac MRI (cMRI)
Measured: Up to 10 years
Number of Participants Experiencing Serious ALI and Acute Liver Failure (ALF)
Measured: Baseline through Month 12
Number of Participants Experiencing Complications Associated with ALI and ALF
Measured: Up to 10 years
Survival Time
Measured: Up to 10 years
Time to First Vertebral Body (Spine) Fracture
Measured: Up to 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Feb 2024
- Study start7 Feb 2024
- Primary completion31 Dec 2028 (estimated)
- Study completion31 Dec 2038 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by Sarepta Therapeutics, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 500 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06270719
- Sponsor's protocol ID
- SRP-9001-401
- Acronym
- ENDURE
- Status last verified
- Oct 2026
- First submitted
- 24 Jan 2024
- Last updated
- 23 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No