32
Participants
Start Date
October 18, 2022
Primary Completion Date
February 19, 2025
Study Completion Date
March 31, 2031
DTX301
non-replicating, self-complementary recombinant adeno-associated virus serotype 8 (AAV8) vector
Placebo
normal saline infusion
Oral Corticosteroids
Participants who receive DTX301 solution will receive oral corticosteroids.
Placebo for oral corticosteroids
Participants who receive Placebo will receive placebo corticosteroids to maintain the study blind
Sodium Acetate
A tracer for the Ureagenesis Rate Test (URT)
Erasmus Universitair Medisch Centrum Rotterrdam, Rotterdam
University Hospitals Cleveland Medical Center, Cleveland
Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago
Universitatsklinikum Heidelberg, Heidelberg
Hopital Femme Mere Enfant, Bron
Necker-Enfants Maladas Hospital, Paris
University of Colorado, Aurora
University of California, Los Angeles
Hospital Italiano de Buenos Aires, Buenos Aires
Clinica Universitaria Reina Fabiola, Córdoba
Hospital de Clinicas de Porto Alegre, Porto Alegre
The Hospital for Sick Children, Toronto
Kumamoto University Hospital, Kumamoto
Fujita Health University Hospital, Toyoake
Centro Hospitalar Universitario de Sao Joao, Porto
Fundacio Hospital Universitari Vall D'Hebron-Institute de Recerca, Barcelona
Ultragenyx Pharmaceutical Inc
INDUSTRY