82
Participants
Start Date
September 27, 2021
Primary Completion Date
March 31, 2029
Study Completion Date
March 31, 2034
UX701
Nonreplicating, recombinant gene transfer vector
Standard of Care (SOC)
SOC treatment (i.e., copper chelators and/or zinc) administered according to standard regimens.
Duke University Medical Center, Durham
University Hospitals Cleveland Medical Center, Cleveland
Indiana University, Indianapolis
University of Michigan, Ann Arbor
Northwestern University, Chicago
University of Utah, Salt Lake City
University of California Los Angeles, Los Angeles
Stanford University, Redwood City
Seattle Children's Hospital, Seattle
University of California Davis, Sacramento
Massachusetts General Hospital, Boston
Vanderbilt University Medical Center, Nashville
Gordon and Leslie Diamond Health Care Centre, Vancouver
Centro Hospitalar Universitário Lisboa Norte, Lisbon
Centro Hospitalar Universitário de São João, Porto
Hospital Universitario Vall d'Hebron - PPDS, Barcelona
Kings College NHS Foundation, London
Ultragenyx Pharmaceutical Inc
INDUSTRY