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Clinical Trials in the USA / NCT06270719
Enrolling by invitation Observational

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

NCT06270719 · tracked via the Priya Life Science USA tracker
Sponsor
Sarepta Therapeutics, Inc.
Phase
Observational
Started
2024-02-07
Last updated
2026-05-04

Condition(s) studied

Duchenne Muscular Dystrophy

Investigational drug(s) / intervention(s)

Delandistrogene MoxeparvovecStandard of Care

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

Standard of Care: No study medication will be provided by the sponsor during this study.

Study 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.

Eligibility

Sex
MALE
Min age
4 Years
Max age
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (25)

FacilityCityRegionStatus
Arkansas Children's Hospital Little Rock Arkansas
Children's Hospital Los Angeles - PIN Los Angeles California
University of Colorado - PPDS Aurora Colorado
Connecticut Children's Medical Center - Hartford Hartford Connecticut
Children's National Medical Center Washington D.C. District of Columbia
Nicklaus Children's Hospital Miami Florida
Nemours Children's Hospital - Orlando Orlando Florida
All Children's Research Institute, Inc St. Petersburg Florida
Ann and Robert H Lurie Childrens Hospital of Chicago Chicago Illinois
Indiana Clinical and Translational Science Institute Indianapolis Indiana
University of Kansas Medical Center Kansas City Kansas
University of Michigan Ann Arbor Michigan
Duke Lenox Baker Children's Hospital Durham North Carolina
Childrens Hospital Medical Center of Akron Akron Ohio
OHSU Healthcare (Oregon Health and Science University) Portland Oregon
Penn State Health Milton S. Hershey Medical Center Hershey Pennsylvania
Children's Hospital of Philadelphia Philadelphia Pennsylvania
Le Bonheur Children's Hospital Memphis Tennessee
Vanderbilt University Medical Center Nashville Tennessee
Cook Children's Hospital Fort Worth Texas
University of Virginia Charlottesville Virginia
Children's Hospital of the King's Daughters Norfolk Virginia
Seattle Children's Hospital Seattle Washington
The Board of Regents of the University of Wisconsin Madison Wisconsin
The Medical College of Wisconsin Milwaukee Wisconsin

More Sarepta Therapeutics, Inc. trials in the USA

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06270719 on ClinicalTrials.gov ↗ ← All trials in the USA