226
Participants
Start Date
July 31, 2019
Primary Completion Date
October 30, 2021
Study Completion Date
October 30, 2021
UCL Cliniques Universitaires Saint-Luc, Brussels
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, Milan
Lysosomal and Rare Disorders Research and Treatment Center, Fairfax
Quirónsalud, Zaragoza
Azienda Ospedaliero Universitaria Meyer, Florence
Mayo Clinic - Rochester, Rochester
Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago
SphinCS GmbH, Höchheim
AP-HP - Hôpitaux Universitaires Est Parisien, Paris
Hospital de Clínicas de Porto Alegre - HCPA, Porto Alegre
Centro Hospitalar Universitario Lisboa Norte, EPE, Lisbon
Centro Universitario Hospitalar de São João, EPE, Porto
Hospital Sant Joan de Deu, Barcelona
Universitätsspital Bern Inselspital, Bern
Universitäts-Kinderspital Zürich, Zurich
University Hospitals Birmingham NHS Foundation Trust, Birmingham
Great Ormond Street Hospital for Children NHS Found. Trust, London
Lead Sponsor
Idorsia Pharmaceuticals Ltd.
INDUSTRY