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

Phase 3

• Conditions: Duchenne Muscular Dystrophy
• Interventions: Genetic: delandistrogene moxeparvovec

• Registration Number: NCT05967351
• Lead Sponsor: Sarepta Therapeutics, Inc.

Brief Summary

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. Pre-infusion baseline will be defined as the timepoint just prior to infusion of delandistrogene moxeparvovec from a previous clinical study. Each participant will be followed for a minimum of 5 years post-infusion of delandistrogene moxeparvovec from a previous clinical study. The duration of participation in this study is dependent on the length of follow-up the participant completed post-infusion of delandistrogene moxeparvovec from a previous clinical study.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-07-21
• Study First Submitted QC: 2023-07-21
• Study First Posted: 2023-08-01
• Study Start: 2023-09-27
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-15
• Last Update Posted: 2025-01-17
• Primary Completion: 2030-11-30
• Study Completion: 2030-11-30

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: Male
• Target Recruitment: 400

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

• Participant or family does not want to disclose participation with general practitioner/primary care physician and other medical providers.

Other inclusion/exclusion criteria may apply.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Delandistrogene Moxeparvovec	delandistrogene moxeparvovec	Participant received delandistrogene moxeparvovec in a previous clinical study.

Primary Outcome Measures

Name	Time	Method
Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)	Up to 5 years after dosing

Secondary Outcome Measures

Name	Time	Method
Change in Time to Rise From Floor From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec	Baseline, 5 years
Change in the Time of 10-meter Walk/Run (10MWR) From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec	Baseline, 5 years
Change in Forced Vital Capacity Percent (FVC%) Predicted From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec	Baseline, 5 years
Change in Peak Expiratory Flow Percent (PEF%) Predicted From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec	Baseline, 5 years
Change in Cardiac Magnetic Resonance Imaging (MRI) Findings From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec	Baseline, 5 years
Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec	Baseline, 5 years
Change in Performance of Upper Limb (PUL) (Version 2.0) Total Scores From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec	Baseline, 5 years
Change in Musculoskeletal MRI Findings From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec	Baseline, 5 years
Change in PUL (Version 2.0) Domain Specific Scores From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec	Baseline, 5 years

Trial Locations

Locations (37)

Oxford University Hospitals NHS Foundation Trust; 🇬🇧 Oxford, Oxfordshire, United Kingdom

Arkansas Children's Hospital; 🇺🇸 Little Rock, Arkansas, United States

UCLA Medical Center; 🇺🇸 Los Angeles, California, United States

Lucile Packard Children's Hospital Stanford (LPCH); 🇺🇸 Palo Alto, California, United States

University of California, Davis; 🇺🇸 Sacramento, California, United States

Rady Children's Hospital; 🇺🇸 San Diego, California, United States

Children's Hospital of Colorado; 🇺🇸 Aurora, Colorado, United States

University of Florida Clinical Research Center; 🇺🇸 Gainesville, Florida, United States

Ann & Robert H. Lurie Children's Hospital of Chicago; 🇺🇸 Chicago, Illinois, United States

University of Iowa ICTS Clinical Research Unit; 🇺🇸 Iowa City, Iowa, United States

The Johns Hopkins Hospital, Rubenstein Child Health Bldg; 🇺🇸 Baltimore, Maryland, United States

Boston Children's Hospital; 🇺🇸 Boston, Massachusetts, United States

Washington University School of Medicine; 🇺🇸 Saint Louis, Missouri, United States

Columbia University Pediatric Neuromuscular Center; 🇺🇸 New York, New York, United States

University of Rochester Medical Center; 🇺🇸 Rochester, New York, United States

Lenox Baker Children's Hospital (Duke University); 🇺🇸 Durham, North Carolina, United States

Nationwide Children's Hospital; 🇺🇸 Columbus, Ohio, United States

Oregon Health and Science University; 🇺🇸 Portland, Oregon, United States

Chiildren's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

Children's Health Specialty Center; 🇺🇸 Dallas, Texas, United States

University of Utah Health; 🇺🇸 Salt Lake City, Utah, United States

Children's Hospital of The King's Daughters; 🇺🇸 Norfolk, Virginia, United States

Children's Hospital Wisconsin; 🇺🇸 Milwaukee, Wisconsin, United States

Universitair Ziekenhuis Gent, Neuromuscular Reference Centre (NMRC); 🇧🇪 Gent, East Flanders, Belgium

Universitätsklinikum Essen - Klinik für Kinderheilkunde I; 🇩🇪 Essen, Nordrhein-Westfalen, Germany

Hong Kong Children's Hospital; 🇭🇰 Hong Kong, Hong Kong

UOC Neurologia Pediatrica e Malattie Muscolari, Istituto G. Gaslini, Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico; 🇮🇹 Genova, Italy

UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; 🇮🇹 Milano, Italy

UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS, Universita Cattoclica del Sacro Cuore; 🇮🇹 Roma, Italy

Kobe University Hospital; 🇯🇵 Kobe, Hyogo, Japan

National Center of Neurology and Psychiatry; 🇯🇵 Kodaira, Tokyo, Japan

Hospital Sant Joan de Deu; 🇪🇸 Esplugues de Llobregat, Barcelona, Spain

Hospital Universitario y Politecnico La fe. Neurology Department; 🇪🇸 Valencia, Spain

Kaohsiung Medical University Chung-Ho Memorial Hospital; 🇨🇳 Kaohsiung, Taiwan

National Taiwan University Hospital; 🇨🇳 Taipei, Taiwan

Great Ormond Street Hospital For Children NHS Foundation Trust; 🇬🇧 London, Greater London, United Kingdom

Institute of Translational and Clinical Research; 🇬🇧 Newcastle Upon Tyne, United Kingdom