NCT06606340

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

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

• Status: Enrolling By Invitation
• Phase: —
• Study type: Observational
• Enrollment: 300 (estimated)
• Sponsor: Sarepta Therapeutics, Inc. · Industry
• Sex: Male
• Age: —
• Healthy volunteers: Not accepted

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. Participants in this study will have been prescribed eteplirsen, golodirsen, or casimersen commercially prior to entry into the study.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
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.
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.
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.

Timeline

• Start date: 2019-01-07
• Primary completion: 2033-12-31
• Completion: 2033-12-31
• First posted: 2024-09-23
• Last updated: 2025-09-10

Locations

20 sites across 1 country: United States

Regulatory

• FDA-regulated drug study