Duchenne Muscular DystrophyEVOLVENCT06606340
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
An observational study of Duchenne Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 20 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Eteplirsen, Golodirsen and Casimersen
- How many people
- 300 participants
- Where
- 20 sitesUnited States
- Study dates
- 7 Jan 2019to 31 Dec 2033
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
20 sites in 1 country. Contact the study team or a site near you to ask about taking part.
20 sites in 1 country
United States20 sites
Kennedy Krieger Institute
Baltimore, Maryland, 21287
UVA Children's Hospital
Charlottesville, Virginia, 22908
Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Nationwide Children's Hospital
Columbus, Ohio, 43205
Duke Lenox Baker Children's Hospital
Durham, North Carolina, 27705
Cook Children's Hospital
Fort Worth, Texas, 76104
University of Florida
Gainesville, Florida, 32610
Penn State Health Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033-0850
Texas Children's Hospital
Houston, Texas, 77030
University Of Iowa Hospitals And Clinics
Iowa City, Iowa, 52242
Arkansas Children's Hospital Research Institute
Little Rock, Arkansas, 72202
Children's Hospital Los Angeles
Los Angeles, California, 90027
Columbia University Medical Center - PIN
New York, New York, 10032
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15213
University of California Davis Medical Center
Sacramento, California, 95817
Seattle Children's Hospital
Seattle, Washington, 98105
Washington University
St Louis, Missouri, 63110
Children's National Hospital
Washington D.C., District of Columbia, 20010
Atrium Health Wake Forest University Health Sciences
Winston-Salem, North Carolina, 27157
About this study
Official 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
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.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Eteplirsen, Golodirsen, Casimersen
- Keywords
- Duchenne Muscular Dystrophy, DMD, Post-marketing, Observational, Phase 4, Eteplirsen, Golodirsen, Casimersen
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EteplirsenDrug
Also called AVI-4658, EXONDYS 51, EXONDYS
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.
GolodirsenDrug
Also called SRP-4053, VYONDYS 53
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.
CasimersenDrug
Also called SRP-4045, AMONDYS 45
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.
| Study group | Role | What they receive |
|---|---|---|
| Eteplirsen | Not described Receives: Drug: Eteplirsen | |
| Golodirsen | Not described Receives: Drug: Golodirsen | |
| Casimersen | Not described Receives: Drug: Casimersen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Loss of Ambulation (LOA)
Measured: Up to 5 years
Time to Rise From the Floor (Supine to Stand)
Measured: Up to 5 years
Time to Walk/Run 10 Meters
Measured: Up to 5 years
Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) Entry Item A
Measured: Up to 5 years
Pulmonary Function, as Measured by Forced Vital Capacity (FVC) (% Predicted)
Measured: Up to 5 years
Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF) as Measured by Echocardiogram (ECHO)
Measured: Up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2024
- Study start7 Jan 2019
- Primary completion31 Dec 2033 (estimated)
- Study completion31 Dec 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by Sarepta Therapeutics, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 300 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06606340
- Sponsor's protocol ID
- 4658-403
- Acronym
- EVOLVE
- Status last verified
- Aug 2025
- First submitted
- 8 Aug 2024
- Last updated
- 10 Sep 2025
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Tian C, Grabich S, Veerapandiyan A, Scharf RJ, Santra S, Hornibrook S, Drummond K, Sehinovych I, Waldrop M. Advancements from the EVOLVE study for assessing real-world experience with eteplirsen, golodirsen and casimersen for the treatment of DMD. J Comp Eff Res. 2026 May;15(5):e250108. doi: 10.57264/cer-2025-0108. Epub 2026 Apr 10.
PubMed 41961051
Study at a glance
From the registry record, last updated by the sponsor on 10 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT06606340
- Acronym / sponsor ID
- EVOLVE / 4658-403
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne Muscular Dystrophy, DMD, Post-marketing, Observational, Phase 4, Eteplirsen, Golodirsen, Casimersen
- Study type
- Observational
- Recruitment status
- Enrolling by invitation
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- Sarepta Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 7 Jan 2019 (actual)
- Primary completion
- 31 Dec 2033 (estimated)
- Study completion
- 31 Dec 2033 (estimated)
- First posted
- 23 Sep 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Eteplirsen | – | Drug: Eteplirsen |
| Golodirsen | – | Drug: Golodirsen |
| Casimersen | – | Drug: Casimersen |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (3 listed)
- 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 (7 listed)
- 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:
- Other inclusion/exclusion criteria may apply.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EteplirsenAlso: AVI-4658, EXONDYS 51, EXONDYSNo 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. | Drug | Eteplirsen |
| GolodirsenAlso: SRP-4053, VYONDYS 53No 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. | Drug | Golodirsen |
| CasimersenAlso: SRP-4045, AMONDYS 45No 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. | Drug | Casimersen |
Primary outcome
Loss of Ambulation (LOA)
Time frame: Up to 5 years
Time to Rise From the Floor (Supine to Stand)
Time frame: Up to 5 years
Time to Walk/Run 10 Meters
Time frame: 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
Pulmonary Function, as Measured by Forced Vital Capacity (FVC) (% Predicted)
Time frame: Up to 5 years
Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF) as Measured by Echocardiogram (ECHO)
Time frame: Up to 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Sep 2025.
- Derived from this study: PubMed 41961051Tian C, Grabich S, Veerapandiyan A, Scharf RJ, Santra S, Hornibrook S, Drummond K, Sehinovych I, Waldrop M. Advancements from the EVOLVE study for assessing real-world experience with eteplirsen, golodirsen and casimersen for the treatment of DMD. J Comp Eff Res. 2026 May;15(5):e250108. doi: 10.57264/cer-2025-0108. Epub 2026 Apr 10.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
The record lists no contact. Use the official record to reach the sponsor.
20 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Duchenne Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 20 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 10 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Loss of Ambulation (LOA) (time frame: Up to 5 years). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06606340, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Sep 2025.