A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Genetic: delandistrogene moxeparvovecGenetic: placebo
- Registration Number
- NCT05881408
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Brief Summary
The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- Male
- Target Recruitment
- 148
- Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
- Cohort 1 only: Non-ambulatory per protocol-specified criteria.
- Cohort 2 only: Ambulatory per protocol-specified criteria and ≥8 to <18 years of age at the time of Screening.
- Ability to cooperate with motor assessment testing.
- Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).
- Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74) antibody titers are not elevated as per protocol-specified requirements.
- A pathogenic frameshift mutation or premature stop codon in the DMD gene, except for any deletion mutations in exon 8 and/or 9.
- Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol specified time limits.
- Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
- Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer.
Other inclusion or exclusion criteria could apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo followed by Delandistrogene Moxeparvovec delandistrogene moxeparvovec Participants will receive matching placebo IV infusion on Day 1. Then, participants will have the opportunity to receive a single IV infusion of delandistrogene moxeparvovec at approximately 72 weeks. Placebo followed by Delandistrogene Moxeparvovec placebo Participants will receive matching placebo IV infusion on Day 1. Then, participants will have the opportunity to receive a single IV infusion of delandistrogene moxeparvovec at approximately 72 weeks. Delandistrogene Moxeparvovec followed by Placebo placebo Participants will receive single IV infusion of delandistrogene moxeparvovec on Day 1. Then, participants will receive a single IV infusion of matching placebo at approximately 72 weeks. Delandistrogene Moxeparvovec followed by Placebo delandistrogene moxeparvovec Participants will receive single IV infusion of delandistrogene moxeparvovec on Day 1. Then, participants will receive a single IV infusion of matching placebo at approximately 72 weeks.
- Primary Outcome Measures
Name Time Method Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72 Baseline, Week 72
- Secondary Outcome Measures
Name Time Method Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72 Baseline, Week 72 Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72 Baseline, Week 72 Part 1 (For Cohort 2 Only): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 72 Baseline, Week 72 Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72 Baseline, Week 72 Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot Week 12 Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72 Baseline, Week 72 Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE) Baseline up to Week 124 Part 1: Change From Baseline in the Middle Domain Score of PUL (Version 2.0) at Week 72 Baseline, Week 72
Trial Locations
- Locations (43)
Gangnam Severance Hospital, Yonsei University Health System
🇰🇷Seoul, Korea, Republic of
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
Lucile Packard Children's Hospital Stanford
🇺🇸Palo Alto, California, United States
University of California at Davis Medical Center
🇺🇸Sacramento, California, United States
Rady Children's Hospital-San Diego
🇺🇸San Diego, California, United States
University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases
🇺🇸Gainesville, Florida, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
University of Iowa Hospitals and Clinics, Dept of Pediatrics
🇺🇸Iowa City, Iowa, United States
The Johns Hopkins Hospital, Charlotte R. Bloomberg Children's Center, Pediatric Clinical Research Unit
🇺🇸Baltimore, Maryland, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Washington University of St. Louis, St. Louis Children's Hospital
🇺🇸Saint Louis, Missouri, United States
University of Rochester, Department of Neurology
🇺🇸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
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Children's Hospital of the King's Daughters
🇺🇸Norfolk, Virginia, United States
The Children's Hospital at Westmead
🇦🇺Westmead, New South Wales, Australia
The Royal Children's Hospital
🇦🇺Parkville, Victoria, Australia
Universitair Ziekenhuis Gent
🇧🇪Gent, Oost-Vlaanderen, Belgium
LMU- Klinikum der Universitat Munchen, Kinderklinik und Kinderpoliklinik im Dr. von Haunerschen Kinderspital, Abeteilung Neuropadiatrie, Campus Innenstadt
🇩🇪Munchen, Bayern, Germany
Universitatsklinikum Essen, Klinik fur Kinderheilkunde I, Abteilung Neuropadiatrie Essen
🇩🇪Essen, Nordrhein-westfalen, Germany
Universitatsklinikum Hamburg Eppendorf
🇩🇪Hamburg, Germany
Hong Kong Children's Hospital
🇭🇰Hong Kong, Hong Kong
Institute of Neruology, Schneider Children's Medical Center of Israel
🇮🇱Petach Tikva, Israel
Tel Aviv Sourasky Medical Center
🇮🇱Tel Aviv, Israel
U.O.S.D Centro Traslazionale di Miologia e Patologie Neurodegenerative, 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
🇮🇹Roma, Italy
National Hospital Organization Osaka Toneyama Medical Center
🇯🇵Toyonaka-shi, Osaka, Japan
National Center of Neurology and Psychiatry
🇯🇵Kodaira, Tokyo, Japan
Pusan National University Yangsan Hospital
🇰🇷Yangsan, Gyeongsangnam-do, Korea, Republic of
Tokyo Women's Medical University Hospital
🇯🇵Shinjuku-ku, Tokyo, Japan
Kyungpook National University Hospital
🇰🇷Daegu, Korea, Republic of
Hospital Sant Joan de Deu
🇪🇸Esplugues de LLobregat, Barcelona, Spain
Sahlgrenska Universitetssjukhuset
🇸🇪Goteborg, Sweden
Karolinska Universitetssjukhuset/Astrid Lindgrens Barnsjukhus, Barnneurologen
🇸🇪Solna, Sweden
Seoul National University Hospital
🇰🇷Seoul, NAP, Korea, Republic of
Hospital Universitari Politecnic La Fe
🇪🇸Valencia, Spain
Kaohsiung Medical University Chung-Ho Memorial Hospital
🇨🇳Kaohsiung, Taiwan
National Taiwan University Hospital
🇨🇳Taipei City, Taiwan
Great Ormond Street Hospital for Children Foundation Trust
🇬🇧London, Greater London, United Kingdom
Oxford University Hospitals NHS Foundation Trust
🇬🇧Oxford, Oxfordshire, United Kingdom
Institute of Translational and Clinical Research
🇬🇧Newcastle Upon Tyne, United Kingdom