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

Официальное название

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

Обзор

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.

Вмешательства

• Препарат 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.
• Препарат 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.
• Препарат 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.

Первичные конечные точки

• Loss of Ambulation (LOA) [Срок оценки: Up to 5 years]
• Time to Rise From the Floor (Supine to Stand) [Срок оценки: Up to 5 years]
• Time to Walk/Run 10 Meters [Срок оценки: Up to 5 years]
• Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) Entry Item A [Срок оценки: Up to 5 years]
• Pulmonary Function, as Measured by Forced Vital Capacity (FVC) (% Predicted) [Срок оценки: Up to 5 years]
• Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF) as Measured by Echocardiogram (ECHO) [Срок оценки: Up to 5 years]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

США · 20 центров

• Arkansas Children's Hospital Research Institute — Little Rock
• Children's Hospital Los Angeles — Los Angeles
• University of California Davis Medical Center — Sacramento
• Children's National Hospital — Washington D.C.
• University of Florida — Gainesville
• University Of Iowa Hospitals And Clinics — Iowa City
• Kennedy Krieger Institute — Baltimore
• Washington University — St Louis
• … и ещё 12 центров

Публикации

• 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. PMID 41961051

Идентификаторы

NCT: NCT06606340 · 4658-403

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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