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
Early research (Phase 1)WithdrawnNCT01003912What this trial is testingFetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage DiseasesWho this might be right forLysosomal Storage DiseasesInborn Errors of Metabolism Joanne Kurtzberg, MD
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
Early research (Phase 1)Not Yet RecruitingNCT07479953What this trial is testingPrenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical TrialWho this might be right forGM1 GangliosidosesGM1 Gangliosidosis, Type IGM1 Gangliosidosis, Type 2 Tippi Mackenzie 5
Testing effectiveness (Phase 2)Ended earlyNCT01372228What this trial is testingPhase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic DisordersWho this might be right forHurler Syndrome (MPS I)Hurler-Scheie SyndromeHunter Syndrome (MPS II)+9 more Talaris Therapeutics Inc. 3
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
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
Not applicableUnknownNCT06539169What this trial is testingFLOWER: Following Longitudinal Outcomes With Epidemiology for Rare DiseasesWho this might be right forAlpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more xCures 1,000
Not applicableUnknownNCT04624789What this trial is testingRegistry GangliosidosesWho this might be right forGangliosidosesGM1 GangliosidosisMorquio B Disease+6 more SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt) 40
Testing effectiveness (Phase 2)Study completedNCT01102686What this trial is testingPyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)Who this might be right forGangliosidoses, GM2Sandhoff DiseaseTay-Sachs Disease The Hospital for Sick Children 20
Not applicableStudy completedNCT04310163What this trial is testingInterviews and Video Capture in Patients With GM1 GangliosidosisWho this might be right forGM1 Gangliosidosis LYSOGENE 25
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
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
Testing effectiveness (Phase 2)Active Not RecruitingNCT04798235What this trial is testingFirst-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 GangliosidosisWho this might be right forInfantile GM2 Gangliosidosis (Disorder) Dr. Anupam Sehgal 3
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 applicableUnknownNCT04320329What this trial is testingNatural History of Morquio B and Late-Onset of GM1 GangliosidosisWho this might be right forMorquio B DiseaseGM1 Gangliosidosis Type III University of British Columbia 30
Not applicableActive Not RecruitingNCT06614569What this trial is testingLong-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff DiseaseWho this might be right forGM2 GangliosidosisTay Sachs DiseaseSandhoff Disease Terence Flotte 7
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
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)Study completedNCT00383448What this trial is testingHSCT for High Risk Inherited Inborn ErrorsWho this might be right forAdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+6 more Masonic Cancer Center, University of Minnesota 38