A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis
Conditions
Niemann-Pick Type C Disease · GM1 Gangliosidosis · GM2 Gangliosidosis
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Locations
UCSF Children's Hospital and Research Center at Oakland, Oakland, California, United States
Contact: Principal Investigator (Caroline Hastings, MD)University of Minnesota Medical School, Minneapolis, Minnesota, United States
Mayo Clinic Childrens Center - PIN, Rochester, Minnesota, United States
Children's Medical Center Dallas, Dallas, Texas, United States
Lysosomal Rare Disorders Research and Treatment Center, Fairfax, Virginia, United States
Hospital Universitario Austral, Ciudad Autónoma Buenos Aires, Buenos Aires, Argentina
Hospital de Niños de La Santisima Trinidad, Córdoba, Córdoba Province, Argentina
Women's and Children's Hospital, North Adelaide, South Australia, Australia
Royal Melbourne Hospital, Parkville, Victoria, Australia
Royal Children's Hospital Melbourne - PIN, Parkville, Victoria, Australia
Instituto Fernandes Figueira, Rio de Janeiro, Rio de Janeiro, Brazil
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS, Porto Alegre, Rio Grande do Sul, Brazil
Contact: Principal Investigator (Roberto Giugliani)Hospital Pequeno Príncipe, Curitiba, Brazil
M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary, Calgary, Alberta, Canada
University of Alberta Medical Genetics Clinic, Edmonton, Alberta, Canada
Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis, Montreal, Quebec, Canada
AP-HP - Hôpital Armand Trousseau, Paris, France
SphinCS GmbH, Höchheim, Germany
Amrita Institute of Medical Sciences and Research Centre, Ernākulam, Kerala, India
Contact: Study coordinator (Sheela Nampoothiri) · sheelanampoothiri@aims.amrita.edu · +91 9447978222All India Institute of Medical Sciences (AIIMS) - New Delhi, New Delhi, National Capital Territory of Delhi, India
JK Lone Hospital, Jaipur, Rajasthan, India
Christian Medical College and Hospital, Vellore, Tamil Nadu, India
Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy
Hospital Universitario Dr. Jose Eleuterio González, Monterrey, Nuevo León, Mexico
Centenario Hospital Miguel Hidalgo, Aguascalientes, Mexico
The Children's Hospital & the University of Child Health Lahore, Lahore, Punjab Province, Pakistan
ULS de Santo António, EPE - Centro Materno Infantil Norte, Porto, Porto District, Portugal
Contact: Study coordinator (Anabela Bandeira) · anabelabandeira.dca@chporto.min-saude.pt · +35 915676520ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS, Lisbon, Portugal
Hospital Universitario Vall d'Hebron - PPDS, Barcelona, Barcelona, Spain
Hospital Infantil Universitario Niño Jesus - PIN, Madrid, Madrid, Spain
Sahlgrenska universitetssjukhuset Östra, Gothenburg, Västra Götaland County, Sweden
Inselspital - Universitätsspital Bern, Bern, Canton of Bern, Switzerland
Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi, Adana, Adana, Turkey (Türkiye)
Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi, Çankaya, Ankara, Turkey (Türkiye)
Ege Universitesi Tip Fakultesi, Bornova, İzmir, Turkey (Türkiye)
Great Ormond Street Hospital, London, London, United Kingdom
University College London Hospitals (UCLH), London, Middlesex, United Kingdom
Royal Manchester Children's Hospital, Manchester, United Kingdom