Not applicableActive Not RecruitingNCT05526664What this trial is testingOmics Gaucher Study: Multiomic ApproachWho this might be right forGaucher Disease CENTOGENE GmbH Rostock 25
Post-approval studies (Phase 4)Study completedNCT04656600What this trial is testingStudy to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type ⅢWho this might be right forGaucher's Disease Sanofi 12
Post-approval studies (Phase 4)Study completedNCT06019728What this trial is testingA Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of FabrazymeWho this might be right forFabry's Disease Sanofi 8
Testing effectiveness (Phase 2)Study completedNCT02004704What this trial is testingA Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase DeficiencyWho this might be right forSphingomyelin Lipidosis Genzyme, a Sanofi Company 25
Not applicableEnded earlyNCT07455864What this trial is testingLysosomal Acid Lipase Deficiency in Risk GroupsWho this might be right forLysosomal Acid Lipase Deficiency AstraZeneca 25
Large-scale testing (Phase 3)WithdrawnNCT05815004What this trial is testingAn Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3Who this might be right forGaucher Disease, Type 3 AVROBIO
Not applicableLooking for participantsNCT05698901What this trial is testingBiomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry DiseaseWho this might be right forFabry Disease Mackay Memorial Hospital 150
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 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
Not applicableUnknownNCT05816993What this trial is testingAssessment of Neurological Manifestations in Gaucher Disease PatientsWho this might be right forGaucher Disease Assiut University 45
Testing effectiveness (Phase 2)Study completedNCT00730314What this trial is testingUnrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood CellsWho this might be right forSickle Cell DiseaseThalassemiaAnemia+9 more Children's Hospital Los Angeles 25
Not applicableStudy completedNCT06801288What this trial is testingDiagnostic Role of Renal Biopsy in Patients With Fabry DiseaseWho this might be right forFabry Disease IRCCS Azienda Ospedaliero-Universitaria di Bologna 80
Large-scale testing (Phase 3)Looking for participantsNCT06904261What this trial is testingMigalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA VariantsWho this might be right forFabry Disease Amicus Therapeutics 8
Testing effectiveness (Phase 2)Study completedNCT02107846What this trial is testingAn Open-Label, Dose Escalation Study to Evaluate the Safety and the Pharmacokinetics of Oral PRX-112Who this might be right forGaucher Disease Protalix 10
Not applicableStudy completedNCT04845958What this trial is testingA Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged SpleenWho this might be right forGaucher Disease, SplenomegalyAcid SphingoMyelinase Deficiency Sanofi 60
Testing effectiveness (Phase 2)Study completedNCT01560182What this trial is testingGene Therapy for Metachromatic Leukodystrophy (MLD)Who this might be right forLysosomal Storage DiseaseMetachromatic Leukodystrophy Orchard Therapeutics 20
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
Not applicableNot Yet RecruitingNCT03725670What this trial is testingDirect Lentiviral Injection Gene Therapy for MLDWho this might be right forMetachromatic Leukodystrophy (MLD) Shenzhen Geno-Immune Medical Institute 10
Not applicableStudy completedNCT04050137What this trial is testingTherapeutic Exercise to Treat Neuropathic PainWho this might be right forGaucher DiseaseFabry Disease Universidad de Zaragoza 5
Testing effectiveness (Phase 2)Study completedNCT01488097What this trial is testingExtension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase DeficiencyWho this might be right forCholesterol Ester Storage Disease (CESD)Lysosomal Acid Lipase DeficiencyLAL-Deficiency Alexion Pharmaceuticals, Inc. 8