Not applicableStudy completedNCT00343577What this trial is testingAntiproteinuric Agents and Fabry DiseaseWho this might be right forFabry DiseaseProteinuria University of Alabama at Birmingham 12
Not applicableStudy completedNCT04997772What this trial is testingDigital Health Platform Customized for Patients With Gaucher DiseaseWho this might be right forGaucher DiseasePatient Reported Outcome Shoshana Vilk 80
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
Testing effectiveness (Phase 2)Ended earlyNCT04273269What this trial is testingA Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 GangliosidosisWho this might be right forGM1 Gangliosidosis LYSOGENE 5
Not applicableStudy completedNCT00351156What this trial is testingStudy to Evaluate Blood Cell Lines From Patients With Gaucher DiseaseWho this might be right forGaucher Disease Amicus Therapeutics 50
Early research (Phase 1)Study completedNCT02422654What this trial is testingTaste Evaluation of Different Liquid Formulations With EliglustatWho this might be right forGaucher Disease Genzyme, a Sanofi Company 8
Not applicableLooking for participantsNCT04613089What this trial is testingNatural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD DatabaseWho this might be right forNeuronal Ceroid LipofuscinosisBatten DiseaseCLN1 Disease+12 more Universitätsklinikum Hamburg-Eppendorf 500
Testing effectiveness (Phase 2)Active Not RecruitingNCT05228145What this trial is testingGene Therapy Study for Children With CLN5 Batten DiseaseWho this might be right forNeuronal Ceroid Lipofuscinosis CLN5 Neurogene Inc. 6
Not applicableLooking for participantsNCT05473637What this trial is testingTaiwan Associated Genetic and Nongenetic Small Vessel DiseaseWho this might be right forCerebral Small Vessel DiseasesCadasilHTRA1-Related Autosomal Dominant Cerebral Angiopathy+5 more National Taiwan University Hospital 500
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
Not applicableUnknownNCT05719337What this trial is testingMulti-Modality Echocardiographic Techniques in Pathological Left Ventricular Hypertrophy AdultsWho this might be right forLeft Ventricular Hypertrophy First Hospital of China Medical University 660
Testing effectiveness (Phase 2)Active Not RecruitingNCT03770572What this trial is testingGene Therapy for Children With CLN3 Batten DiseaseWho this might be right forCLN3Batten Disease Neela Therapeutics 7
Large-scale testing (Phase 3)Study completedNCT05529992What this trial is testingVelaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher DiseaseWho this might be right forGaucher Disease Takeda 20
Testing effectiveness (Phase 2)Study completedNCT03406962What this trial is testingMGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)Who this might be right forInherited Metabolic Disorders (IMD) Magenta Therapeutics, Inc. 8
Post-approval studies (Phase 4)Study completedNCT04120506What this trial is testingLong Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)Who this might be right forGaucher Disease, Type 1 Shaare Zedek Medical Center 15
Large-scale testing (Phase 3)Study completedNCT00074971What this trial is testingThe Safety and Efficacy of Fabrazyme in Patients With Fabry DiseaseWho this might be right forFabry Disease Genzyme, a Sanofi Company 58
Testing effectiveness (Phase 2)Ended earlyNCT03454893What this trial is testingOpen Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry DiseaseWho this might be right forFabry Disease AVROBIO 15
Not applicableUnknownNCT03542370What this trial is testingUmbrella Review of the DASH Dietary Pattern and Cardiometabolic RiskWho this might be right forCardiovascular Risk FactorCardiovascular DiseasesCoronary Heart Disease+6 more University of Toronto 1
Early research (Phase 1)Study completedNCT02800070What this trial is testingAutologous Stem Cell Transplantation of Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry DiseaseWho this might be right forFabry Disease University Health Network, Toronto 5
Not applicableWithdrawnNCT01093105What this trial is testingAn Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (GLD)Who this might be right forLeukodystrophy, Globoid Cell Shire