Not applicableUnknownNCT04624789What this trial is testingRegistry GangliosidosesWho this might be right forGangliosidosesGM1 GangliosidosisMorquio B Disease+6 more SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt) 40
Large-scale testing (Phase 3)WithdrawnNCT02921620What this trial is testingStudy to Evaluate the Safety and EffIcacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry DiseaseWho this might be right forFabry Disease Protalix
Testing effectiveness (Phase 2)Study completedNCT04046224What this trial is testingDose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)Who this might be right forFabry Disease Sangamo Therapeutics 36
Not applicableStudy completedNCT02372513What this trial is testingNational Lysosomal Acid Lipase Deficiency StudyWho this might be right forCholesteryl Ester Storage Disease Ankara University 810
Not applicableUnknownNCT02120235What this trial is testingInvestigating Lysosomal Storage Diseases in Minority GroupsWho this might be right forLysosomal Storage DisordersGaucher DiseaseFabry Disease+2 more O & O Alpan LLC 20,000
Testing effectiveness (Phase 2)UnknownNCT00004293What this trial is testingPhase II Study of Glucocerebrosidase in Patients With Gaucher DiseaseWho this might be right forGaucher's Disease National Center for Research Resources (NCRR) 24
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 applicableStudy completedNCT01971957What this trial is testingSjogren-Larsson Syndrome: Natural History, Clinical Variation and Evaluation of Biochemical MarkersWho this might be right forSjogren-Larsson Syndrome (SLS) University of Nebraska 20
Not applicableUnknownNCT04758130What this trial is testingGetting Global Rare Disease Insights Through Technology StudyWho this might be right forMetabolic DiseaseFabry DiseaseGaucher Disease+2 more M.A.G.I.C. Clinic LTD 150
Post-approval studies (Phase 4)Study completedNCT00364858What this trial is testingSafety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher DiseaseWho this might be right forGaucher Disease, Type 1Cerebroside Lipidosis SyndromeGlucocerebrosidase Deficiency Disease+2 more Genzyme, a Sanofi Company 95
Not applicableLooking for participantsNCT06941025What this trial is testingMaternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® in Women With Fabry Disease and Their Infants During Pregnancy and BreastfeedingWho this might be right forFabry DiseasePregnancyPregnancy Complications Chiesi Farmaceutici S.p.A. 10
Post-approval studies (Phase 4)Study completedNCT00074984What this trial is testingThe Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry DiseaseWho this might be right forFabry Disease Genzyme, a Sanofi Company 82
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 applicableUnknownNCT04833595What this trial is testingCan Mindful Eating Improve Cholesterol and Glucose LevelWho this might be right forCholesterol; Lipidosis Monash University Malaysia 60
Not applicableStudy completedNCT01019629What this trial is testingFabry Screening StudyWho this might be right forFabry Disease Baylor Research Institute 2,724
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 completedNCT00975689What this trial is testingBiomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl CysteineWho this might be right forNiemann-Pick Disease, Type C Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 35
Testing effectiveness (Phase 2)Study completedNCT00358150What this trial is testingThe Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher PatientsWho this might be right forGaucher Disease, Type 1Cerebroside Lipidosis SyndromeGlucocerebrosidase Deficiency Disease+2 more Genzyme, a Sanofi Company 26
Testing effectiveness (Phase 2)Study completedNCT03950050What this trial is testingAmbroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement TherapyWho this might be right forGaucher Disease, Type 1 Shaare Zedek Medical Center 40
Not applicableEnded earlyNCT04073888What this trial is testingStudy of the Spermatic Characteristics of Patients With Fabry DiseaseWho this might be right forFabry Disease University Hospital, Bordeaux 22