75
Participants
Start Date
June 30, 2025
Primary Completion Date
November 4, 2027
Study Completion Date
November 4, 2027
AZ-3102
Nizubaglustat
Placebo
Placebo
NOT_YET_RECRUITING
Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi, Adana
NOT_YET_RECRUITING
Inselspital - Universitätsspital Bern, Bern
NOT_YET_RECRUITING
Royal Children's Hospital Melbourne - PIN, Parkville
NOT_YET_RECRUITING
Women's and Children's Hospital, North Adelaide
NOT_YET_RECRUITING
Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi, Çankaya
RECRUITING
Hospital Universitario Vall d'Hebron - PPDS, Barcelona
NOT_YET_RECRUITING
AP-HM- Hôpital de La Timone, Marseille
RECRUITING
Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan
RECRUITING
Hospital Infantil Universitario Niño Jesus - PIN, Madrid
NOT_YET_RECRUITING
Ege Universitesi Tip Fakultesi, Bornova
NOT_YET_RECRUITING
Mayo Clinic Children's Center - PIN, Rochester
NOT_YET_RECRUITING
AP-HP - Hôpital Armand Trousseau, Paris
NOT_YET_RECRUITING
Children's Hospital and Research Center at Oakland, Oakland
NOT_YET_RECRUITING
All India Institute of Medical Sciences (AIIMS) - New Delhi, New Delhi
NOT_YET_RECRUITING
JK Lone Hospital, Jaipur
NOT_YET_RECRUITING
Seth G S Medical College and K E M Hospital, Mumbai
NOT_YET_RECRUITING
Amrita Institute of Medical Sciences and Research Centre, Ernākulam
NOT_YET_RECRUITING
Lysosomal Rare Disorders Research and Treatment Center, Fairfax
NOT_YET_RECRUITING
Hospital Universitario Austral, Ciudad Autónoma Buenos Aires
NOT_YET_RECRUITING
Hospital de Niños de La Santisima Trinidad, Córdoba
NOT_YET_RECRUITING
Instituto Fernandes Figueira, Rio de Janeiro
NOT_YET_RECRUITING
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS, Porto Alegre
NOT_YET_RECRUITING
Hospital Pequeno Principe, Curitiba
NOT_YET_RECRUITING
Great Ormond Street Hospital NHSFT, London
NOT_YET_RECRUITING
M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary, Calgary
NOT_YET_RECRUITING
University of Alberta Medical Genetics Clinic, Edmonton
NOT_YET_RECRUITING
Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis, Montreal
RECRUITING
ULS de Santo António, EPE - Centro Materno Infantil Norte, Porto
RECRUITING
ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS, Lisbon
RECRUITING
Sahlgrenska universitetssjukhuset Östra, Gothenburg
NOT_YET_RECRUITING
University College London Hospitals (UCLH), London
Lead Sponsor
Azafaros A.G.
INDUSTRY