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

NCT06270719 | Enrolling By Invitation FDA Drug

• 1 other identifier: SRP-9001-401
• Study Type: observational
• Target: 500
• Locations: 1 country
• Sites: 25

Brief 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 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

Keywords

Duchenne Muscular Dystrophy; DMD; Observational; Standard of Care; Phase 4; ELEVIDYS

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (11)

• 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

Study Arms (2)

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

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

Interventions

Delandistrogene Moxeparvovec GENETIC

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

Also known as: SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS

Cohort 1 (Treated)

Standard of Care DRUG

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

Also known as: Chronic glucocorticoid treatment

Cohort 2 (Standard of Care)

Eligibility Criteria

• Age: 4 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Sarepta Therapeutics, Inc.: lead

Study Sites (25)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location

Children's Hospital Los Angeles - PIN

Los Angeles, California, 90027, United States

Location

University of Colorado - PPDS

Aurora, Colorado, 80045, United States

Location

Connecticut Children's Medical Center - Hartford

Hartford, Connecticut, 06106, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

Location

Nemours Children's Hospital - Orlando

Orlando, Florida, 32827, United States

Location

All Children's Research Institute, Inc

St. Petersburg, Florida, 33701, United States

Location

Ann and Robert H Lurie Childrens Hospital of Chicago

Chicago, Illinois, 60611-2991, United States

Location

Indiana Clinical and Translational Science Institute

Indianapolis, Indiana, 46202, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Duke Lenox Baker Children's Hospital

Durham, North Carolina, 27704, United States

Location

Childrens Hospital Medical Center of Akron

Akron, Ohio, 44302, United States

Location

OHSU Healthcare (Oregon Health and Science University)

Portland, Oregon, 97239, United States

Location

Penn State Health Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Le Bonheur Children's Hospital

Memphis, Tennessee, 38103, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

Cook Children's Hospital

Fort Worth, Texas, 76104, United States

Location

University of Virginia

Charlottesville, Virginia, 22903, United States

Location

Children's Hospital of the King's Daughters

Norfolk, Virginia, 23507, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

The Board of Regents of the University of Wisconsin

Madison, Wisconsin, 53715-1218, United States

Location

The Medical College of Wisconsin

Milwaukee, Wisconsin, 53226-4874, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

Standard of Care

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Quality Indicators, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 24, 2024
• First Posted: February 21, 2024
• Study Start: February 7, 2024
• Primary Completion (Estimated): December 31, 2029
• Study Completion (Estimated): December 31, 2038
• Last Updated: May 4, 2026

Record last verified: 2026-04

Locations

US (25)