A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
NCT ID: NCT06606340
Last Updated: 2025-09-10
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ENROLLING_BY_INVITATION
300 participants
OBSERVATIONAL
2019-01-07
2033-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Eteplirsen
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.
Golodirsen
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.
Casimersen
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.
Interventions
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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.
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.
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.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Has an established clinical diagnosis of DMD, as documented prior to screening by a genetic report.
* Receiving, or initiating treatment with, eteplirsen, golodirsen, or casimersen at the time of observational study enrollment. Note: Participants with a prescription for eteplirsen, golodirsen, or casimersen at enrollment must initiate the exon-skipping therapy within 6 months of the date of enrollment or will no longer be eligible for this study. Note: Enrollment of eteplirsen participants has been completed, no additional participants will be enrolled.
Exclusion Criteria
* Has declined to provide consent for collection of their genetic data.
* Has a medical condition or confounding circumstances that, in the opinion of the Investigator, might compromise:
1. The participant's ability to comply with the protocol-required procedures
2. The participant's wellbeing or safety, and/or
3. The clinical interpretability of the data collected from the participant.
MALE
No
Sponsors
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Sarepta Therapeutics, Inc.
INDUSTRY
Responsible Party
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Locations
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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
Countries
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Other Identifiers
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4658-403
Identifier Type: -
Identifier Source: org_study_id
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