129
Participants
Start Date
December 3, 2024
Primary Completion Date
July 31, 2026
Study Completion Date
November 30, 2027
GTX-102
antisense oligonucleotide
Sham-LP
Small needle prick on the lower back at the location where the LP injection is normally made
Columbia University Medical Center, New York
Universitaetsklinikum Hamburg-Eppendorf, Hamburg
Rare Disease Research, Hillsborough
UNC Chapel Hill Pediatrics, Chapel Hill
Hospital Universitario Puerta de Hierro, Madrid
Rare Disease Research, Atlanta
Nicklaus Children's Hospital, Miami
Hospital Universitario Virgen del Rocio, Seville
Rush University, Chicago
Children's Mercy, Kansas City
Carum Research Inc, Dallas
The University of Texas, Austin
Children's Hospital Colorado, Aurora
Medical University of Gdańsk, Gdansk
Haunersche Kinderklinik, Munich
Cedars Sinai, Los Angeles
UCSD, Rady Children's Hospital, San Diego
UCSF, San Francisco
Boston Children's Hospital, Boston
McGill University Health Centre, Montreal
British Columbia Children's Hospital, Vancouver
University of Leipzig, Leipzig
Nagoya City University Graduate School of Medical Sciences, Nagoya
Osaka City General Hospital, Osaka
Hokkaido University Hospital, Sapporo
Polish Mothers Memorial Institute, Lodz
Hospital Universitario Parc Tauli, Barcelona
Hospital Sant Joan de Deu, Barcelona
Ultragenyx Pharmaceutical Inc
INDUSTRY