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

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

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.

Study Overview

• Status: Enrolling by invitation
• Conditions: Duchenne Muscular Dystrophy
• Intervention / Treatment: Drug: Eteplirsen; Drug: Golodirsen; Drug: Casimersen

• Study Type: Observational
• Enrollment (Estimated): 300

Contacts and Locations

Study Locations

• United States
  • Arkansas
    • Little Rock, Arkansas, United States, 72202
      • Arkansas Children's Hospital Research Institute
  • California
    • Los Angeles, California, United States, 90027
      • Children's Hospital Los Angeles
    • Sacramento, California, United States, 95817
      • University of California Davis Medical Center
  • District of Columbia
    • Washington D.C., District of Columbia, United States, 20010
      • Children's National Hospital
  • Florida
    • Gainesville, Florida, United States, 32610
      • University of Florida
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa Hospitals and Clinics
  • Maryland
    • Baltimore, Maryland, United States, 21287
      • Kennedy Krieger Institute
  • Missouri
    • St Louis, Missouri, United States, 63110
      • Washington University
  • New York
    • New York, New York, United States, 10032
      • Columbia University Medical Center - PIN
  • North Carolina
    • Durham, North Carolina, United States, 27705
      • Duke Lenox Baker Children's Hospital
    • Winston-Salem, North Carolina, United States, 27157
      • Atrium Health Wake Forest University Health Sciences
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Children's Hospital Medical Center
    • Columbus, Ohio, United States, 43205
      • Nationwide Children's Hospital
  • Pennsylvania
    • Hershey, Pennsylvania, United States, 17033-0850
      • Penn State Health Milton S. Hershey Medical Center
    • Philadelphia, Pennsylvania, United States, 19104
      • Children's Hospital of Philadelphia
    • Pittsburgh, Pennsylvania, United States, 15213
      • UPMC Children's Hospital of Pittsburgh
  • Texas
    • Fort Worth, Texas, United States, 76104
      • Cook Children's Hospital
    • Houston, Texas, United States, 77030
      • Texas Children's Hospital
  • Virginia
    • Charlottesville, Virginia, United States, 22908
      • UVA Children's Hospital
  • Washington
    • Seattle, Washington, United States, 98105
      • Seattle Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

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.

Description

Key Inclusion Criteria:

• 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.

Key Exclusion Criteria:

• 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:

  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.

Other inclusion/exclusion criteria may apply.

Study Plan

How is the study designed?

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Eteplirsen	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.; Other Names: AVI-4658; EXONDYS 51; EXONDYS
Golodirsen	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.; Other Names: SRP-4053; VYONDYS 53
Casimersen	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.; Other Names: SRP-4045; AMONDYS 45

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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

Collaborators and Investigators

• Sponsor: Sarepta Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): January 7, 2019
• Primary Completion (Estimated): December 31, 2033
• Study Completion (Estimated): December 31, 2033

Study Registration Dates

• First Submitted: August 8, 2024
• First Submitted That Met QC Criteria: September 19, 2024
• First Posted (Actual): September 23, 2024

Study Record Updates

• Last Update Posted (Estimated): September 10, 2025
• Last Update Submitted That Met QC Criteria: September 3, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Keywords: Observational; Duchenne Muscular Dystrophy; DMD; Phase 4; Eteplirsen; Post-marketing; Golodirsen; Casimersen
• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Muscular Disorders, Atrophic; Muscular Dystrophies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Muscular Dystrophy, Duchenne; eteplirsen; golodirsen; casimersen
• Other Study ID Numbers: 4658-403

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes