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NCT06606340

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

Enrolling by Invitation
Not specifiedAll AgesObservational
Sarepta Therapeutics, Inc.
~300 participants
Updated 2025-09-10 on ClinicalTrials.gov
What's tested:EteplirsenGolodirsenCasimersen

At a glance

Recruiting sites
0 of 20 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Loss of Ambulation (LOA)
Measured over Up to 5 years
+5 more outcomes measured

Conditions

Duchenne Muscular Dystrophy

Explore related trials

Duchenne Muscular DystrophyDMD Gene

Where it's being run

20 sites across 14 states
Pennsylvania3
California2
North Carolina2
Ohio2
Texas2
Arkansas1
District of Columbia1
Florida1

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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.

Exclusion

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:

What this trial measures

  • 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 MetersUp to 5 years
  • Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) Entry Item AUp 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