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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 GangliosidosisBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, GM1 gangliosidosis, GM2 gangliosidosis, Gangliosidoses, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosisActive, not recruitingPhase 32026-09-22NCT07082543

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OrganizationsLinked trials
Azafaros B.V.5

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Research sitesCityCountryLinked trials
All India Institute of Medical Sciences (AIIMS) - New DelhiNew DelhiIndia5
Amrita Institute of Medical Sciences and Research CentreErnākulamIndia3
AP-HP - Hôpital Armand TrousseauParisFrance3
AP-HP - Hôpital de la Pitié SalpétrièreParisFrance2
Balcali Hastanesi Saglik Uygulama ve Arastirma MerkeziAdanaTurkey (Türkiye)1
Children's Medical Center DallasDallasUnited States18
Ege Universitesi Tip FakultesiBornovaTurkey (Türkiye)3
Fondazione IRCCS Istituto Neurologico Carlo BestaMilanItaly35
Gazi Universitesi Saglik Arastirma ve Uygulama MerkeziÇankayaTurkey (Türkiye)3
Hospital de Clinicas de Porto Alegre (HCPA) - PPDSPorto AlegreBrazil5
Hospital de Niños de La Santisima TrinidadCórdobaArgentina3
Hospital Infantil Universitario Niño Jesus - PINMadridSpain3
Hospital Universitario AustralCiudad Autónoma Buenos AiresArgentina2
Hospital Universitario Vall d'Hebron - PPDSBarcelonaSpain4
Inselspital - Universitätsspital BernBernSwitzerland4
Instituto Fernandes FigueiraRio de JaneiroBrazil3
JK Lone HospitalJaipurIndia3
Lysosomal Rare Disorders Research and Treatment CenterFairfaxUnited States3
M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in CalgaryCalgaryCanada3
Mayo Clinic Children's Center - PINRochesterUnited States2
Sahlgrenska universitetssjukhuset ÖstraGothenburgSweden2
SphinCS GmbHHöchheimGermany5
UCSF Children's Hospital and Research Center at OaklandOaklandUnited States3
ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDSLisbonPortugal4
ULS de Santo António, EPE - Centro Materno Infantil NortePortoPortugal3