Status Sponsors Study Title NCT #
(link)		 Intervention Study Type Conditions
NCT07054515AZA-001-301RECRUITINGGM2 Gangliosidosis AzafarosINTERVENTIONAL
NCT06906367AT1001-030XRECRUITINGFabry Disease   
NCT06663358CLI-06657AA1-10RECRUITINGFabry Disease   
NCT06858397A proof-of Concept Study to Assess Safety and Tolerability of HM15321/GC1134A in Patients With Fabry DiseaseRECRUITINGFabry DiseaseDRUG: HM15421/GC1134AGC Biopharma CorpINTERVENTIONAL
NCT071231552405N1221RECRUITINGPompe Disease   
NCT05487599A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)RECRUITINGGaucher Disease| Gaucher Disease, Type 1GENETIC: LY3884961Prevail TherapeuticsINTERVENTIONAL
NCT06904261AT1001-033RECRUITINGFabry Disease   
NCT06121011POM-005RECRUITINGPompe Disease   
NCT04860960Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1ACTIVE NOT RECRUITINGNiemann-Pick Disease, Type C1DRUG: Hydroxypropyl-beta-cyclodextrin| DRUG: PlaceboCyclo Therapeutics, Inc.INTERVENTIONAL
NCT02843035Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy ExtensionACTIVE NOT RECRUITINGGaucher Disease Type 1| Gaucher Disease Type 3DRUG: venglustat (GZ402671)|DRUG: imigluceraseGenzyme, a Sanofi CompanyINTERVENTIONAL
NCT06545136FLT201-02ACTIVE NOT RECRUITINGGaucher DiseaseGENETIC: FLT201Freeline TherapeuticsINTERVENTIONAL
NCT06270316CT-AMT-191-01ACTIVE NOT RECRUITINGFabry Disease  INTERVENTIONAL
NCT06109948ABX1100-1001ACTIVE NOT RECRUITINGPompe Disease  INTERVENTIONAL
NCT05206773A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry DiseaseACTIVE NOT RECRUITINGFabry DiseaseDRUG: Venglustat (GZ402671)| DRUG: PlaceboSanofiINTERVENTIONAL
NCT06150820AT845-02ACTIVE NOT RECRUITINGPompe Disease  INTERVENTIONAL
NCT05222906Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3ACTIVE NOT RECRUITINGGaucher Disease Type 3DRUG: Venglustat|DRUG: imigluceraseSanofiINTERVENTIONAL
NCT05039866ST-920-LT01ACTIVE NOT RECRUITINGFabry DiseaseBIOLOGICAL: ST-920Sangamo TherapeuticsINTERVENTIONAL
NCT05280548A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry DiseaseACTIVE NOT RECRUITINGFabry DiseaseDRUG: Venglustat (GZ402671) |DRUG: Agalsidase alfa |DRUG: Agalsidase beta (GZ419828) |DRUG: MigalastatSanofiINTERVENTIONAL
NCT03737214A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry DiseaseACTIVE NOT RECRUITINGFabry DiseaseDRUG: LucerastatIdorsia Pharmaceuticals Ltd.INTERVENTIONAL
NCT07223944FLT201-03NOT YET RECRUITINGGaucher Disease Freeline TherapeuticsINTERVENTIONAL
NCT06328608CLI-06657AA1-01NOT YET RECRUITINGFabry Disease   
NCT07354724DNLI-J-0001NOT YET RECRUITINGPompe Disease   
NCT0200031013-CFCT-07RECRUITING  Lysosomal and Rare Disorders Research and Treatment Center, Inc.OBSERVATIONAL
NCT03812055Cellular Pharmacodynamics of Small Molecules in Lysosomal Storage DisordersRECRUITINGLysosomal Storage Diseases Lysosomal and Rare Disorders Research and Treatment Center, Inc.OBSERVATIONAL
NCT05705674The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3)RECRUITINGSanfilippo Disease Lysosomal and Rare Disorders Research and Treatment Center, Inc.OBSERVATIONAL
NCT0661489424-LDRTC-01RECRUITING  Lysosomal and Rare Disorders Research and Treatment Center, Inc.OBSERVATIONAL
Not yet on CT.govFD24-LDRTC-03RECRUITINGFabry Disease Lysosomal and Rare Disorders Research and Treatment Center, Inc. 
Not yet on CT.govGD25-LDRTC-01RECRUITINGGaucher Disease Lysosomal and Rare Disorders Research and Treatment Center, Inc. 
Not yet on CT.govPD25-LDRTC-02RECRUITINGPompe Disease Lysosomal and Rare Disorders Research and Treatment Center, Inc. 
NCT0606560523-LDRTC-01ACTIVE NOT RECRUITING  Lysosomal and Rare Disorders Research and Treatment Center, Inc.OBSERVATIONAL
NCT0611607123-LDRTC-02ACTIVE NOT RECRUITING  Lysosomal and Rare Disorders Research and Treatment Center, Inc.OBSERVATIONAL
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