Not applicableLooking for participantsNCT03333200What this trial is testingLongitudinal Study of Neurodegenerative DisordersWho this might be right forMLDKrabbe DiseaseALD+24 more University of Pittsburgh 1,500
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 completedNCT04041102What this trial is testingNatural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) PatientsWho this might be right forGM1 Gangliosidosis University of Pennsylvania 31
Not applicableStudy completedNCT02699190What this trial is testingLeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for LeukodystrophiesWho this might be right forLeukodystrophyWhite Matter Disease4H Syndrome+61 more Children's Hospital of Philadelphia 236
Not applicableStudy completedNCT00006057What this trial is testingDiagnostic and Screening Study of Genetic DisordersWho this might be right forTay-Sachs DiseasePorphyria, ErythropoieticLeukodystrophy, Globoid Cell+1 more National Center for Research Resources (NCRR) 50
Early research (Phase 1)Ended earlyNCT04669535What this trial is testingA Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff DiseaseWho this might be right forTay-Sachs DiseaseSandhoff Disease Terence Flotte 9
Early research (Phase 1)WithdrawnNCT00679744What this trial is testingA Phase I Study of Pyrimethamine in Patients With GM2 GangliosidosisWho this might be right forG(M2) GangliosideTay-Sachs Disease GangliosideSandhoff Disease Ganglioside Exsar Corporation
Large-scale testing (Phase 3)Ended earlyNCT04395339What this trial is testingGM1 Prophylaxis for WBRT Related Cognitive DysfunctionWho this might be right forBrain MetastasesRadiotherapy Side EffectCognitive Impairment+2 more Sun Yat-sen University 51
Not applicableStudy completedNCT04310163What this trial is testingInterviews and Video Capture in Patients With GM1 GangliosidosisWho this might be right forGM1 Gangliosidosis LYSOGENE 25
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
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
Large-scale testing (Phase 3)Looking for participantsNCT07082543What 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 A.G. 75
Testing effectiveness (Phase 2)Looking for participantsNCT03952637What this trial is testingIntravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 GangliosidosisWho this might be right forLysosomal DiseasesGangliosidosisGM1 National Human Genome Research Institute (NHGRI) 54
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
Not applicableActive Not RecruitingNCT02851862What this trial is testingA Natural History of Late Onset Tay-Sachs DiseaseWho this might be right forGM2 Gangliosidosis Massachusetts General Hospital 10
Not applicableStudy completedNCT04084301What this trial is testingImpact of Cardiopulmonary Bypass Flow on Renal Oxygenation, Blood Flow and Tubular InjuryWho this might be right forExtracorporeal Circulation; ComplicationsRenal FailureCirculation Disorder+4 more Sahlgrenska University Hospital 100
Not applicableStudy completedNCT01999257What this trial is testingEfficacy Study of an Online Educational Module Before Carrier Genetic Screening in Persons of Ashkenazi Jewish Descent.Who this might be right forTay Sachs DiseaseCanavan DiseaseFamilial Dysautonomia McGill University Health Centre/Research Institute of the McGill University Health Centre 60
Large-scale testing (Phase 3)Ended earlyNCT00654433What this trial is testingALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic DiseasesWho this might be right forInherited Metabolic DiseasesLysosomal Storage DisordersPeroxisomal Storage Diseases+2 more Aldagen 40
Testing effectiveness (Phase 2)Active Not RecruitingNCT04713475What this trial is testingStudy of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 GangliosidosisWho this might be right forGM1 GangliosidosisGM1 Gangliosidosis, Type IGM1 Gangliosidosis, Type 2+1 more Gemma Biotherapeutics 26
Large-scale testing (Phase 3)Looking for participantsNCT07082725What this trial is testingEvaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)Who this might be right forNiemann-Pick Type C Disease Azafaros A.G. 72