Phase
Condition
Muscular Dystrophy
Treatment
delandistrogene moxeparvovec
placebo
Clinical Study ID
Male
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Definitive diagnosis of DMD based on documented clinical findings and prior genetictesting.
Cohort 1 only: Non-ambulatory per protocol-specified criteria.
Cohort 2 only: Ambulatory per protocol-specified criteria and ≥8 to <18 years of ageat 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 formodifications to accommodate changes in weight).
Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74) antibody titers are notelevated as per protocol-specified requirements.
A pathogenic frameshift mutation or premature stop codon in the DMD gene, except forany deletion mutations in exon 8 and/or 9.
Exclusion
Exclusion Criteria:
Exposure to gene therapy, investigational medication, or any treatment designed toincrease 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, orrequirement for chronic drug treatment that in the opinion of the Investigatorcreates unnecessary risk for gene transfer.
Other inclusion or exclusion criteria could apply.
Study Design
Connect with a study center
The Children's Hospital at Westmead
Westmead, New South Wales 2145
AustraliaSite Not Available
The Royal Children's Hospital
Parkville, Victoria 3052
AustraliaSite Not Available
Universitair Ziekenhuis Gent
Gent, Oost-Vlaanderen 9000
BelgiumSite Not Available
The Children's Hospital of Eastern Ontario
Ottawa, Ontario K1H8L1
CanadaSite Not Available
Research Institute McGill University Health Centre
Montréal, Quebec H4A3J1
CanadaSite Not Available
Centre Hospitalier Universitaire de Québec - Université Laval (pavillon Centre Hospitalier Universitaire Laval)
Québec, G1V 4G2
CanadaSite Not Available
LMU- Klinikum der Universitat Munchen, Kinderklinik und Kinderpoliklinik im Dr. von Haunerschen Kinderspital, Abeteilung Neuropadiatrie, Campus Innenstadt
Munchen, Bayern
GermanySite Not Available
Universitatsklinikum Essen, Klinik fur Kinderheilkunde I, Abteilung Neuropadiatrie Essen
Essen, Nordrhein-westfalen
GermanySite Not Available
Universitatsklinikum Hamburg Eppendorf
Hamburg,
GermanySite Not Available
Hong Kong Children's Hospital
Hong Kong,
Hong KongSite Not Available
Institute of Neruology, Schneider Children's Medical Center of Israel
Petach Tikva,
IsraelSite Not Available
Tel Aviv Sourasky Medical Center
Tel Aviv, 6423906
IsraelSite Not Available
U.O.S.D Centro Traslazionale di Miologia e Patologie Neurodegenerative, Istituto G. Gaslini, Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico
Genova, 16147
ItalySite Not Available
IRCCS Istituto Neurologico Carlo Besta Neurepsichiatria Infantile 2 - Epilettologia e Neurologia dello Sviluppo
Milano, 20133
ItalySite Not Available
UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milano, 20122
ItalySite Not Available
UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS
Roma, 00168
ItalySite Not Available
National Hospital Organization Osaka Toneyama Medical Center
Toyonaka-shi, Osaka 560-8552
JapanSite Not Available
National Center of Neurology and Psychiatry
Kodaira, Tokyo 187-8551
JapanSite Not Available
Tokyo Women's Medical University Hospital
Shinjuku-ku, Tokyo 162-866
JapanSite Not Available
Pusan National University Yangsan Hospital
Yangsan, Gyeongsangnam-do 50612
Korea, Republic ofSite Not Available
Seoul National University Hospital
Seoul, NAP 03080
Korea, Republic ofSite Not Available
Kyungpook National University Hospital
Daegu, 41944
Korea, Republic ofSite Not Available
Gangnam Severance Hospital, Yonsei University Health System
Seoul, 06273
Korea, Republic ofSite Not Available
Hospital Sant Joan de Deu
Esplugues de LLobregat, Barcelona 08950
SpainSite Not Available
Hospital Universitari Politecnic La Fe
Valencia, 46026
SpainSite Not Available
Sahlgrenska Universitetssjukhuset
Goteborg, 416 85
SwedenSite Not Available
Karolinska Universitetssjukhuset/Astrid Lindgrens Barnsjukhus, Barnneurologen
Solna, 171 76
SwedenSite Not Available
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung,
TaiwanSite Not Available
National Taiwan University Hospital
Taipei City,
TaiwanSite Not Available
Great Ormond Street Hospital for Children Foundation Trust
London, Greater London WC1N 3JH
United KingdomSite Not Available
Oxford University Hospitals NHS Foundation Trust
Oxford, Oxfordshire
United KingdomSite Not Available
Institute of Translational and Clinical Research
Newcastle Upon Tyne, NE13BZ
United KingdomSite Not Available
Arkansas Children's
Little Rock, Arkansas 72202
United StatesSite Not Available
Arkansas Children's Hospital
Little Rock, Arkansas 72202
United StatesSite Not Available
Lucile Packard Children's Hospital Stanford
Palo Alto, California 94304
United StatesSite Not Available
University of California at Davis Medical Center
Sacramento, California 95817
United StatesSite Not Available
Rady Children's Hospital-San Diego
San Diego, California 92123
United StatesSite Not Available
University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases
Gainesville, Florida 32608
United StatesSite Not Available
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois 60611
United StatesSite Not Available
University of Iowa Hospitals and Clinics, Dept of Pediatrics
Iowa City, Iowa 52242
United StatesSite Not Available
The Johns Hopkins Hospital, Charlotte R. Bloomberg Children's Center, Pediatric Clinical Research Unit
Baltimore, Maryland 21287
United StatesSite Not Available
Boston Children's Hospital
Boston, Massachusetts 02115
United StatesSite Not Available
Washington University of St. Louis, St. Louis Children's Hospital
Saint Louis, Missouri 63110
United StatesSite Not Available
University of Rochester, Department of Neurology
Rochester, New York 14642
United StatesSite Not Available
University of Rochester, Dept of Neurology
Rochester, New York 14642
United StatesSite Not Available
Lenox Baker Children's Hospital (Duke University)
Durham, North Carolina 27705
United StatesSite Not Available
Nationwide Children's Hospital
Columbus, Ohio 43205
United StatesSite Not Available
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United StatesSite Not Available
VUMC- Monroe Carell Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee 37203
United StatesSite Not Available
Children's Hospital of the King's Daughters
Norfolk, Virginia 23510
United StatesSite Not Available
Children's Wisconsin
Milwaukee, Wisconsin 53226
United StatesSite Not Available

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