A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
EVOLVE
1 other identifier
observational
300
1 country
20
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Jan 2019
Longer than P75 for all trials
20 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
January 7, 2019
CompletedFirst Submitted
Initial submission to the registry
August 8, 2024
CompletedFirst Posted
Study publicly available on registry
September 23, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 31, 2033
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 31, 2033
September 10, 2025
August 1, 2025
15 years
August 8, 2024
September 3, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (6)
Loss of Ambulation (LOA)
Up to 5 years
Time to Rise From the Floor (Supine to Stand)
Up to 5 years
Time to Walk/Run 10 Meters
Up to 5 years
Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) Entry Item A
Up to 5 years
Pulmonary Function, as Measured by Forced Vital Capacity (FVC) (% Predicted)
Up to 5 years
Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF) as Measured by Echocardiogram (ECHO)
Up to 5 years
Study Arms (3)
Eteplirsen
Golodirsen
Casimersen
Interventions
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.
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.
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.
Eligibility Criteria
Participants within each prescriber's practice who are receiving eteplirsen, golodirsen, or casimersen, in routine clinical practice, and who meet the study eligibility criteria and provide informed consent (either by the participant or through authorization by a legal guardian), will be invited to enroll into the study and will be followed according to the protocol.
You may qualify if:
- Is willing to provide informed assent or consent (if applicable) and has a parent(s) or legal guardian(s) or is a participant ≥18 years of age who is (are) willing to provide informed consent for the participant to participate in the study and comply with study data collection procedures.
- 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.
You may not qualify if:
- Is currently participating in any DMD interventional study at the time of this study enrollment.
- 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:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (20)
Arkansas Children's Hospital Research Institute
Little Rock, Arkansas, 72202, United States
Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
University of California Davis Medical Center
Sacramento, California, 95817, United States
Children's National Hospital
Washington D.C., District of Columbia, 20010, United States
University of Florida
Gainesville, Florida, 32610, United States
University Of Iowa Hospitals And Clinics
Iowa City, Iowa, 52242, United States
Kennedy Krieger Institute
Baltimore, Maryland, 21287, United States
Washington University
St Louis, Missouri, 63110, United States
Columbia University Medical Center - PIN
New York, New York, 10032, United States
Duke Lenox Baker Children's Hospital
Durham, North Carolina, 27705, United States
Atrium Health Wake Forest University Health Sciences
Winston-Salem, North Carolina, 27157, United States
Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Penn State Health Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033-0850, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15213, United States
Cook Children's Hospital
Fort Worth, Texas, 76104, United States
Texas Children's Hospital
Houston, Texas, 77030, United States
UVA Children's Hospital
Charlottesville, Virginia, 22908, United States
Seattle Children's Hospital
Seattle, Washington, 98105, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 8, 2024
First Posted
September 23, 2024
Study Start
January 7, 2019
Primary Completion (Estimated)
December 31, 2033
Study Completion (Estimated)
December 31, 2033
Last Updated
September 10, 2025
Record last verified: 2025-08