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ENROLLING BY INVITATION
NCT06606340

A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice

Sponsor: Sarepta Therapeutics, Inc.

View on ClinicalTrials.gov

Summary

This is a phase 4, multicenter, prospective, observational study designed to collect both medical history data and prospective data on Duchenne Muscular Dystrophy (DMD) treatment outcomes in participants receiving eteplirsen, golodirsen, and casimersen in routine clinical practice. Participants in this study will have been prescribed eteplirsen, golodirsen, or casimersen commercially prior to entry into the study.

Official title: A Long-term Observational Study Evaluating Sarepta Therapeutics, Inc.'s Exon-Skipping Therapies in Patients With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice

Key Details

Gender

MALE

Age Range

Any - Any

Study Type

OBSERVATIONAL

Enrollment

300

Start Date

2019-01-07

Completion Date

2033-12-31

Last Updated

2025-09-10

Healthy Volunteers

No

Interventions

DRUG

Eteplirsen

No study medication will be provided by the Sponsor as a condition to participate in this observational study, and all decisions concerning each participant's treatment are at the discretion of the participant's treating physician. Participants will receive treatment as prescribed by the treating physician as part of standard of care.

DRUG

Golodirsen

No study medication will be provided by the Sponsor as a condition to participate in this observational study, and all decisions concerning each participant's treatment are at the discretion of the participant's treating physician. Participants will receive treatment as prescribed by the treating physician as part of standard of care.

DRUG

Casimersen

No study medication will be provided by the Sponsor as a condition to participate in this observational study, and all decisions concerning each participant's treatment are at the discretion of the participant's treating physician. Participants will receive treatment as prescribed by the treating physician as part of standard of care.

Locations (20)

Arkansas Children's Hospital Research Institute

Little Rock, Arkansas, United States

Children's Hospital Los Angeles

Los Angeles, California, United States

University of California Davis Medical Center

Sacramento, California, United States

Children's National Hospital

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

University of Florida

Gainesville, Florida, United States

University Of Iowa Hospitals And Clinics

Iowa City, Iowa, United States

Kennedy Krieger Institute

Baltimore, Maryland, United States

Washington University

St Louis, Missouri, United States

Columbia University Medical Center - PIN

New York, New York, United States

Duke Lenox Baker Children's Hospital

Durham, North Carolina, United States

Atrium Health Wake Forest University Health Sciences

Winston-Salem, North Carolina, United States

Children's Hospital Medical Center

Cincinnati, Ohio, United States

Nationwide Children's Hospital

Columbus, Ohio, United States

Penn State Health Milton S. Hershey Medical Center

Hershey, Pennsylvania, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Cook Children's Hospital

Fort Worth, Texas, United States

Texas Children's Hospital

Houston, Texas, United States

UVA Children's Hospital

Charlottesville, Virginia, United States

Seattle Children's Hospital

Seattle, Washington, United States