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

September 3, 2025 updated by: Sarepta Therapeutics, Inc.

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

Status

Indicates the current recruitment status or the expanded access status.
Enrolling by invitation

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
300

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

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

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Loss of Ambulation (LOA)
Time Frame: Up to 5 years
Up to 5 years
Time to Rise From the Floor (Supine to Stand)
Time Frame: Up to 5 years
Up to 5 years
Time to Walk/Run 10 Meters
Time Frame: Up to 5 years
Up to 5 years
Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) Entry Item A
Time Frame: Up to 5 years
Up to 5 years
Pulmonary Function, as Measured by Forced Vital Capacity (FVC) (% Predicted)
Time Frame: Up to 5 years
Up to 5 years
Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF) as Measured by Echocardiogram (ECHO)
Time Frame: Up to 5 years
Up to 5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Sarepta Therapeutics, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 7, 2019

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 31, 2033

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 31, 2033

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
August 8, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 19, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
September 23, 2024

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 10, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 3, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Cookie Settings

We use cookies to ensure that we give you the best experience on our website. For more details carefully read our Privacy and Cookies Policy. ...>>>You can change your preferences or withdraw your consent at any time by deleting the cookies from your website or computer as described in the policy. Please accept it and choose your preferences by ticking the corresponding boxes in the "Manage Settings" section below. Please carefully read our Terms of Service before you proceed with using any part of this website.
«Manage Settings