Not applicableStudy completedNCT02851862What this trial is testingA Natural History of Late Onset Tay-Sachs DiseaseWho this might be right forGM2 Gangliosidosis Massachusetts General Hospital 10
Testing effectiveness (Phase 2)Looking for participantsNCT07399704What this trial is testingEvaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C DiseaseWho this might be right forGM2 GangliosidosisNiemann-Pick Type C Disease Azafaros B.V. 21
Not applicableStudy completedNCT01626092What this trial is testingReduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal DisordersWho this might be right forLysosomal Storage DiseasePeroxisomal Disorder Masonic Cancer Center, University of Minnesota 3
Not applicableStudy completedNCT04470713What this trial is testingNatural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2Who this might be right forGM1 GangliosidosisGM2 GangliosidosisGaucher Disease, Type 2+3 more Idorsia Pharmaceuticals Ltd. 226
Large-scale testing (Phase 3)Study completedNCT00672022What this trial is testingPharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral DosesWho this might be right forGM2 GangliosidosesTay-SachsSandhoff Disease Children's National Research Institute 10
Testing effectiveness (Phase 2)Study completedNCT05758922What this trial is testingPhase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C DiseaseWho this might be right forGM2 GangliosidosisNiemann-Pick Disease, Type C Azafaros B.V. 13
Not applicableStudy completedNCT01023048What this trial is testingConcurrent Single Gene and 24 Chromosome Aneuploidy Preimplantation Genetic Diagnosis (PGD)Who this might be right forAny Single Gene Disorder (Cystic Fibrosis, Tay-Sachs) Natera, Inc. 56
Large-scale testing (Phase 3)Active Not RecruitingNCT07082543What this trial is testingEvaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 GangliosidosisWho this might be right forGangliosidoses, GM2Gangliosidosis, GM1 Azafaros B.V. 75
Large-scale testing (Phase 3)Study completedNCT00176904What this trial is testingStem Cell Transplant for Inborn Errors of MetabolismWho this might be right forAdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+8 more Masonic Cancer Center, University of Minnesota 135
Post-approval studies (Phase 4)Ended earlyNCT02030015What this trial is testingSynergistic Enteral Regimen for Treatment of the GangliosidosesWho this might be right forGM1 GangliosidosesGM2 GangliosidosesTay-Sachs Disease+1 more University of Minnesota 16
Not applicableStudy completedNCT05109793What this trial is testingGM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)Who this might be right forGM1 GangliosidosisSandhoff DiseaseTay-Sachs Disease Azafaros B.V. 31
Testing effectiveness (Phase 2)Study completedNCT00418847What this trial is testingPharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 GangliosidosisWho this might be right forGangliosidoses GM2 The Hospital for Sick Children 5
Not applicableLooking for participantsNCT03047369What this trial is testingThe Myelin Disorders Biorepository ProjectWho this might be right forLeukodystrophyWhite Matter DiseaseLeukoencephalopathies+63 more Children's Hospital of Philadelphia 12,000
Large-scale testing (Phase 3)Ended earlyNCT04221451What this trial is testingA Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2Who this might be right forTay-Sachs DiseaseSandhoff Disease Genzyme, a Sanofi Company 75
Not applicableStudy completedNCT01869270What this trial is testingGene Therapy for Tay-Sachs DiseaseWho this might be right forTay Sachs DiseaseSandhoff DiseaseLate Onset Tay Sachs Disease University of Minnesota 4
Not applicableNot Yet RecruitingNCT07445490What this trial is testingTranslational Potential of ex Vivo Gene Therapy in GM2 GangliosidosisWho this might be right forTay-Sachs Disease GangliosideSandhoff Disease Ganglioside Assistance Publique - Hôpitaux de Paris 6
Not applicableLooking for participantsNCT00668187What this trial is testingA Natural History Study of the GangliosidosesWho this might be right forTay-Sachs DiseaseSandhoff DiseaseLate Onset Tay-Sachs Disease+2 more University of Minnesota 52
Early research (Phase 1)Looking for participantsNCT02254863What this trial is testingUCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like CellsWho this might be right forAdrenoleukodystrophyBatten DiseaseMucopolysaccharidosis II+9 more Joanne Kurtzberg, MD 40