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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

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Duchenne Muscular DystrophyDMD Gene

NCT06606340

Where you'd take part

This study runs at 20 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Arkansas Children's Hospital Research Institute

    Little Rock, Arkansasno site contact published

  • Atrium Health Wake Forest University Health Sciences

    Winston-Salem, North Carolinano site contact published

  • Children's Hospital Los Angeles

    Los Angeles, Californiano site contact published

  • Children's Hospital Medical Center

    Cincinnati, Ohiono site contact published

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvaniano site contact published

  • Children's National Hospital

    Washington D.C., District of Columbiano site contact published

  • Columbia University Medical Center - PIN

    New York, New Yorkno site contact published

  • Cook Children's Hospital

    Fort Worth, Texasno site contact published

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Who to contact

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

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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