49
Participants
Start Date
November 8, 2021
Primary Completion Date
February 20, 2024
Study Completion Date
February 28, 2026
DTX401
nonreplicating, recombinant, adeno-associated virus (AAV) serotype 8 (AAV8)
Placebo
Normal Saline infusion
Oral corticosteroids
Participants who receive DTX401 solution will receive oral corticosteroids
Placebo for oral corticosteroids
Participants who receive Placebo will receive placebo oral corticosteroids to maintain the study blind
Righospitalet, Copenhagen
Mount Sinai, The Bronx
Hospital Clinico Universitario de Santiago, Santiago de Compostela
Istituto Giannina Gaslini, Genova
Children's Hospital of Philadelphia, Philadelphia
University Medical Center Eppendorf, Hamburg
Duke University, Durham
Cleveland Clinic, Cleveland
University of Texas, Houston
Children's Hospital Colorado, Aurora
University of Naples, Naples
Primary Children's Hospital, Salt Lake City
Children's Hospital of Orange County, Orange
University of Connecticut Health Center, Farmington
Hospital de Clinicas de Porto Alegre, Porto Alegre
McGill University, Montreal
Kumamoto University Hospital, Kumamoto
Osaka City General Hospital, Osaka
Fujita Health University Hospital, Toyoake
Groningen University, Groningen
Ultragenyx Pharmaceutical Inc
INDUSTRY