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Enrolling by Invitation

NCT Number: NCT06606340

A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in 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.

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Key information

Conditions

Duchenne Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Sex eligibility

Male

Study type

Observational

Primary location

Arkansas Children's Hospital Research Institute, Little Rock, Arkansas, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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:
  • 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.

Other inclusion/exclusion criteria may apply.

Treatment and study plan

Eteplirsen

Drug

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

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

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

Primary outcomes

  1. Loss of Ambulation (LOA)

    Time frame: Up to 5 years

  2. Time to Rise From the Floor (Supine to Stand)

    Time frame: Up to 5 years

  3. Time to Walk/Run 10 Meters

    Time frame: Up to 5 years

  4. Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) Entry Item A

    Time frame: Up to 5 years

  5. Pulmonary Function, as Measured by Forced Vital Capacity (FVC) (% Predicted)

    Time frame: Up to 5 years

  6. Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF) as Measured by Echocardiogram (ECHO)

    Time frame: Up to 5 years

Sponsors and collaborators

Lead sponsor

Sarepta Therapeutics, Inc.

Industry

Registry information

Official study 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

Acronym: EVOLVE

Important dates

Study start
2019
Primary completion
2033
Study completion
2033
First posted
Sep 23, 2024
Registry last updated
Sep 10, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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