Enrolling by invitation NCT05967351
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: delandistrogene moxeparvovec.
- Who it may be relevant to
- Registry conditions: Duchenne Muscular Dystrophy. Basic parameters: No limits · Male.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Belgium, Germany, Hong Kong, Italy +4
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
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
Overview
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.
Interventions
- Genetic 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.
Primary outcome measures
- Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI) [Time frame: Up to 10 years]
Secondary outcome measures (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 [Time frame: 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 [Time frame: 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 [Time frame: 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 [Time frame: 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 [Time frame: 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 [Time frame: 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 [Time frame: 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 [Time frame: Baseline, up to 10 years]
- Change in Musculoskeletal MRI Findings From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation [Time frame: Baseline, up to 10 years]
Eligibility criteria
Inclusion criteria
- 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.
Exclusion criteria
- Not applicable
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Other
Study locations
United States · 22 centers
- 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
- … and 14 more centers
Italy · 3 centers
- 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
Japan · 3 centers
- Kobe University Hospital — Kobe
- National Center of Neurology and Psychiatry — Kodaira
- Tokyo Women's Medical University Hospital — Tokyo
United Kingdom · 3 centers
- 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
Spain · 2 centers
- Hospital Sant Joan de Deu — Esplugues de Llobregat
- Hospital Universitario y Politecnico La fe. Neurology Department — Valencia
Taiwan · 2 centers
- Kaohsiung Medical University Chung-Ho Memorial Hospital — Kaohsiung City
- National Taiwan University Hospital — Taipei
Belgium · 1 center
- Universitair Ziekenhuis Gent, Neuromuscular Reference Centre (NMRC) — Ghent
Germany · 1 center
- Universitätsklinikum Essen - Klinik für Kinderheilkunde I — Essen
Hong Kong · 1 center
- Hong Kong Children's Hospital — Hong Kong
Identifiers
NCT: NCT05967351 · SRP-9001-305 · 2023-505043-39-00