119
Participants
Start Date
February 1, 2024
Primary Completion Date
June 30, 2027
Study Completion Date
June 30, 2027
No Intervention
No investigational drug will be administered to participants in this study.
University of Pittsburgh Medical Center, Pittsburgh
University of Pennsylvania, Philadelphia
Lysosomal and Rare Diseases Research and Treatment Center, Inc., Fairfax
Emory Clinic, Atlanta
University of Cincinnati, Cincinnati
University of Michigan, Ann Arbor
Children's Hospitals and Clinics of Minnesota, Minneapolis
University of Kansas Medical Center, Kansas City
University of UTAH - PPDS, Salt Lake City
University of California Irvine, Irvine
Hackensack University Medical Center, Hackensack
AU61003, Adelaide
AU61001, Herston
BR55003, Flamengo
BR55002, Porto Alegre
BR55001, São Paulo
CN15003, Edmonton
CA15001, Montreal
FR33006, Angers
FR33009, Garches
FR33005, Lille
FR33007, Limoges
FR33002, Marseille
FR33003, Nantes
FR33004, Nice
FR33001, Strasbourg
DT49005, Bonn
DT49004, Essen
DT49003, Höchheim
DT49006, Münster
IT39002, Florence
IT39005, Gussago
IT39012, Messina
IT39009, Milan
IT39011, Milan
IT39008, Pavia
IT39006, Pisa
IT39004, Roma
IT39003, Udine
National Center of Neurology and Psychiatry, Kodaira-Shi
Tokyo Women's Medical University Hospital, Shinjuku-Ku
ES34003, Albacete
ES34004, Barcelona
ES34009, Donostia / San Sebastian
ES34007, L'Hospitalet de Llobregat
ES34001, Madrid
ES34005, Madrid
ES34002, Valencia
TW88601, Taipei
TW88602, Taipei
TW88603, Taoyuan
UK44003, Cambridge
UK44001, Newcastle upon Tyne
UK44004, Salford
Lead Sponsor
Astellas Gene Therapies
INDUSTRY