A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study

Официальное название

A Phase 3, Multinational, Long-term Follow-up Study to Evaluate Safety and Efficacy in Subjects Who Have Previously Received SRP-9001 in a Clinical Study

Обзор

The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received delandistrogene moxeparvovec in a previous clinical study. No study drug will be administered as part of this study.

Вмешательства

• Генная терапия delandistrogene moxeparvovec
  No study drug will be administered as part of this study. Eligible participants who received treatment with delandistrogene moxeparvovec during a previous clinical study will be included.

Первичные конечные точки

• Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI) [Срок оценки: Up to 10 years]

Вторичные конечные точки (9)

• Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation [Срок оценки: Baseline, up to 10 years]
• Change in Time to Rise From Floor From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation [Срок оценки: Baseline, up to 10 years]
• Change in the Time of 10-meter Walk/Run (10MWR) From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation [Срок оценки: Baseline, up to 10 years]
• Change in Performance of Upper Limb (PUL) (Version 2.0) Total Scores From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation [Срок оценки: Baseline, up to 10 years]
• Change in PUL (Version 2.0) Domain Specific Scores From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation [Срок оценки: Baseline, up to 10 years]
• Change in Forced Vital Capacity Percent (FVC%) Predicted From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation [Срок оценки: Baseline, up to 10 years]
• Change in Peak Expiratory Flow Percent (PEF%) Predicted From Pre-infusion Baseline to the End of the Study Participation of Delandistrogene Moxeparvovec [Срок оценки: Baseline, up to 10 years]
• Change in Cardiac Magnetic Resonance Imaging (MRI) Findings From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation [Срок оценки: Baseline, up to 10 years]
• Change in Musculoskeletal MRI Findings From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation [Срок оценки: Baseline, up to 10 years]

Критерии участия

Критерии включения

• Received delandistrogene moxeparvovec for Duchenne muscular dystrophy in a previous clinical study.
• Has (a) parent(s) or legal caregiver(s) or is ≥18 years of age and able to understand and comply with the study visit schedule and all other protocol requirements.

Критерии исключения

• Not applicable

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Другое

Центры проведения

США · 22 центра

• Arkansas Children's Hospital — Little Rock
• UCLA Medical Center — Los Angeles
• Lucile Packard Children's Hospital Stanford (LPCH) — Palo Alto
• University of California, Davis — Sacramento
• Rady Children's Hospital — San Diego
• Children's Hospital of Colorado — Aurora
• University of Florida Clinical Research Center — Gainesville
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago
• … и ещё 14 центров

Италия · 3 центра

• UOC Neurologia Pediatrica e Malattie Muscolari, Istituto G. Gaslini, Istituto Pediatrico d — Genova
• UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico — Milan
• UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universita — Roma

Япония · 3 центра

• Kobe University Hospital — Kobe
• National Center of Neurology and Psychiatry — Kodaira
• Tokyo Women's Medical University Hospital — Tokyo

Великобритания · 3 центра

• Great Ormond Street Hospital For Children NHS Foundation Trust — London
• Oxford University Hospitals NHS Foundation Trust — Oxford
• Institute of Translational and Clinical Research — Newcastle upon Tyne

Испания · 2 центра

• Hospital Sant Joan de Deu — Esplugues de Llobregat
• Hospital Universitario y Politecnico La fe. Neurology Department — Valencia

Тайвань · 2 центра

• Kaohsiung Medical University Chung-Ho Memorial Hospital — Kaohsiung City
• National Taiwan University Hospital — Taipei

Бельгия · 1 центр

• Universitair Ziekenhuis Gent, Neuromuscular Reference Centre (NMRC) — Ghent

Германия · 1 центр

• Universitätsklinikum Essen - Klinik für Kinderheilkunde I — Essen

Гонконг · 1 центр

• Hong Kong Children's Hospital — Гонконг

Идентификаторы

NCT: NCT05967351 · SRP-9001-305 · 2023-505043-39-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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