ENROLLING_BY_INVITATION

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

Conditions

Duchenne Muscular Dystrophy DMD Observational Standard of Care Phase 4

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

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

Quick Facts

Study Start:2024-02-07

Study Completion:2038-12-31

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:ENROLLING_BY_INVITATION

Study ID

NCT06270719

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.

Ages Eligible for Study:4 Years

Sexes Eligible for Study:MALE

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
* Has a definitive diagnosis of DMD prior to Screening based on documentation of clinical findings and confirmatory genetic testing. * Is currently receiving or has been prescribed to start chronic glucocorticoid therapy at the time of this observational study enrollment. * Is at least 4 years of age at the time of enrollment. * Is ambulatory per protocol specified 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 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.

Inclusion Criteria

Exclusion Criteria

* Has a definitive diagnosis of DMD prior to Screening based on documentation of clinical findings and confirmatory genetic testing.
* Is currently receiving or has been prescribed to start chronic glucocorticoid therapy at the time of this observational study enrollment.
* Is at least 4 years of age at the time of enrollment.
* Is ambulatory per protocol specified 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 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.

Contacts and Locations

Study Locations (Sites)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202

United States

University of Colorado - PPDS

Aurora, Colorado, 80045

United States

Children's National Medical Center

Washington, District of Columbia, 20010

United States

Nicklaus Children's Hospital

Miami, Florida, 33155

United States

Nemours Children's Hospital - Orlando

Orlando, Florida, 32827

United States

All Children's Research Institute, Inc

Saint Petersburg, Florida, 33701

United States

Ann and Robert H Lurie Childrens Hospital of Chicago

Chicago, Illinois, 60611-2991

United States

Indiana Clinical and Translational Science Institute

Indianapolis, Indiana, 46202

United States

University of Kansas Medical Center

Kansas City, Kansas, 66160

United States

University of Michigan

Ann Arbor, Michigan, 48109

United States

OHSU Healthcare (Oregon Health and Science University)

Portland, Oregon, 97239

United States

Penn State Health Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033

United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104

United States

Le Bonheur Children's Hospital

Memphis, Tennessee, 38103

United States

Vanderbilt University Medical Center

Nashville, Tennessee, 37232

United States

Cook Children's Hospital

Fort Worth, Texas, 76104

United States

Children's Hospital of the King's Daughters

Norfolk, Virginia, 23507

United States

The Board of Regents of the University of Wisconsin

Madison, Wisconsin, 53715-1218

United States

The Medical College of Wisconsin

Milwaukee, Wisconsin, 53226-4874

United States

Collaborators and Investigators

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 Registration Dates

Study Start Date2024-02-07

Study Completion Date2038-12-31

Study Record Updates

Study Start Date2024-02-07

Study Completion Date2038-12-31

Terms related to this study

Keywords Provided by Researchers

Duchenne Muscular Dystrophy
DMD
Observational
Standard of Care
Phase 4

Additional Relevant MeSH Terms

Duchenne Muscular Dystrophy