A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Elevidys in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) (ENVISION)
Study Overview
- Age
- 8 years and older
- Phase
- Phase 3
- Sponsor
- Sarepta Therapeutics
- Therapeutic Approach
- Gene Therapy
- Variant Requirement
- Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
- Eligible Sex
- Male
- Ambulation
- Ambulatory and Non-Ambulatory
- Study Start (Actual)
- 2023-05-31
- Primary Completion (Estimated)
- 2027-05-31
- Study Completion (Estimated)
- 2028-06-30
- Enrollment (Estimated)
- 148
- Countries
- United StatesAustraliaBelgiumCanadaGermanyHong KongIsraelItalyJapanSouth KoreaSpainSwedenTaiwanUnited Kingdom
Study Requirements and Criteria
Steroid Use
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).
Inclusion Criteria
- 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.
Exclusion Criteria
- 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.
Contacts and Locations
Contacts and Locations
Locations
This study has 46 locations
United States
Arkansas Locations
Little Rock, Arkansas, United States, 72202
Arkansas Children's Hospital
California Locations
Palo Alto, California, United States, 94304
Lucile Packard Children's Hospital Stanford
Sacramento, California, United States, 95817
University of California at Davis Medical Center
San Diego, California, United States, 92123
Rady Children's Hospital-San Diego
Florida Locations
Gainesville, Florida, United States, 32608
University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases
Illinois Locations
Chicago, Illinois, United States, 60611
Ann & Robert H. Lurie Children's Hospital of Chicago
Maryland Locations
Baltimore, Maryland, United States, 21287
The Johns Hopkins Hospital, Charlotte R. Bloomberg Children's Center, Pediatric Clinical Research Unit
Massachusetts Locations
Boston, Massachusetts, United States, 02115
Boston Children's Hospital
Missouri Locations
St Louis, Missouri, United States, 63110
Washington University of St. Louis, St. Louis Children's Hospital
New York Locations
Rochester, New York, United States, 14642
University of Rochester, Department of Neurology
North Carolina Locations
Durham, North Carolina, United States, 27705
Lenox Baker Children's Hospital (Duke University)
Ohio Locations
Columbus, Ohio, United States, 43205
Nationwide Children's Hospital
Pennsylvania Locations
Philadelphia, Pennsylvania, United States, 19104
Children's Hospital of Philadelphia
Virginia Locations
Norfolk, Virginia, United States, 23510
Children's Hospital of the King's Daughters
Australia
New South Wales Locations
Westmead, New South Wales, Australia, 2145
The Children's Hospital at Westmead
Victoria Locations
Parkville, Victoria, Australia, 3052
The Royal Children's Hospital
Belgium
Oost-Vlaanderen Locations
Ghent, Oost-Vlaanderen, Belgium, 9000
Universitair Ziekenhuis Gent
Canada
Ontario Locations
Ottawa, Ontario, Canada, K1H8L1
The Children's Hospital of Eastern Ontario
Quebec Locations
Montreal, Quebec, Canada, H4A3J1
Research Institute McGill University Health Centre
Québec, Canada, G1V 4G2
Centre Hospitalier Universitaire de Québec - Université Laval (pavillon Centre Hospitalier Universitaire Laval)
Germany
Bavaria Locations
München, Bavaria, Germany
LMU- Klinikum der Universitat Munchen, Kinderklinik und Kinderpoliklinik im Dr. von Haunerschen Kinderspital, Abeteilung Neuropadiatrie, Campus Innenstadt
North Rhine-Westphalia Locations
Essen, North Rhine-Westphalia, Germany
Universitatsklinikum Essen, Klinik fur Kinderheilkunde I, Abteilung Neuropadiatrie Essen
Hamburg, Germany
Universitatsklinikum Hamburg Eppendorf
Hong Kong
Hong Kong, Hong Kong
Hong Kong Children's Hospital
Israel
Petah Tikva, Israel
Institute of Neruology, Schneider Children's Medical Center of Israel
Tel Aviv, Israel, 6423906
Tel Aviv Sourasky Medical Center
Italy
Genova, Italy, 16147
U.O.S.D Centro Traslazionale di Miologia e Patologie Neurodegenerative, Istituto G. Gaslini, Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico
Milan, Italy, 20122
UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Italy, 20133
IRCCS Istituto Neurologico Carlo Besta Neurepsichiatria Infantile 2 - Epilettologia e Neurologia dello Sviluppo
Roma, Italy, 00168
UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS
Japan
Osaka Locations
Toyonaka-shi, Osaka, Japan, 560-8552
National Hospital Organization Osaka Toneyama Medical Center
Tokyo Locations
Kodaira, Tokyo, Japan, 187-8551
National Center of Neurology and Psychiatry
Shinjuku-ku, Tokyo, Japan, 162-866
Tokyo Women's Medical University Hospital
South Korea
Gyeongsangnam-do Locations
Yangsan, Gyeongsangnam-do, South Korea, 50612
Pusan National University Yangsan Hospital
NAP Locations
Seoul, NAP, South Korea, 03080
Seoul National University Hospital
Daegu, South Korea, 41944
Kyungpook National University Hospital
Seoul, South Korea, 06273
Gangnam Severance Hospital, Yonsei University Health System
Spain
Barcelona Locations
Esplugues de Llobregat, Barcelona, Spain, 08950
Hospital Sant Joan de Deu
Valencia, Spain, 46026
Hospital Universitari Politecnic La Fe
Sweden
Gothenburg, Sweden, 416 85
Sahlgrenska Universitetssjukhuset
Solna, Sweden, 171 76
Karolinska Universitetssjukhuset/Astrid Lindgrens Barnsjukhus, Barnneurologen
Taiwan
Kaohsiung City, Taiwan
Kaohsiung Medical University Chung-Ho Memorial Hospital
Taipei, Taiwan
National Taiwan University Hospital
United Kingdom
Greater London Locations
London, Greater London, United Kingdom, WC1N 3JH
Great Ormond Street Hospital for Children Foundation Trust
Oxfordshire Locations
Oxford, Oxfordshire, United Kingdom
Oxford University Hospitals NHS Foundation Trust
Newcastle upon Tyne, United Kingdom, NE13BZ
Institute of Translational and Clinical Research
Clinical Trial Registry
NCT ID
NCT05881408This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.