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

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.

Conditions

Duchenne Muscular Dystrophy

Talk to us

Study Overview

On this page

Study Details

Study overview

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.

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

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

Condition
Duchenne Muscular Dystrophy
Intervention / Treatment

-

Contacts and Locations

Little Rock

Arkansas Children's Hospital, Little Rock, Arkansas, United States, 72202

Aurora

University of Colorado - PPDS, Aurora, Colorado, United States, 80045

Washington

Children's National Medical Center, Washington, District of Columbia, United States, 20010

Miami

Nicklaus Children's Hospital, Miami, Florida, United States, 33155

Orlando

Nemours Children's Hospital - Orlando, Orlando, Florida, United States, 32827

Indianapolis

Indiana Clinical and Translational Science Institute, Indianapolis, Indiana, United States, 46202

Kansas City

University of Kansas Medical Center, Kansas City, Kansas, United States, 66160

Ann Arbor

University of Michigan, Ann Arbor, Michigan, United States, 48109

Hershey

Penn State Health Milton S. Hershey Medical Center, Hershey, Pennsylvania, United States, 17033

Philadelphia

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States, 19104

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.

For general information about clinical research, read Learn About Studies.

Eligibility 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.

Ages Eligible for Study

4 Years to

Sexes Eligible for Study

MALE

Accepts Healthy Volunteers

No

Collaborators and Investigators

Sarepta Therapeutics, Inc.,

Study Record Dates

2038-12-31