Status	Sponsors	Study Title	NCT # (link)	Intervention	Study Type	Conditions
NCT07054515	AZA-001-301	RECRUITING	GM2 Gangliosidosis		Azafaros	INTERVENTIONAL
NCT06906367	AT1001-030X	RECRUITING	Fabry Disease
NCT06663358	CLI-06657AA1-10	RECRUITING	Fabry Disease
NCT06858397	A proof-of Concept Study to Assess Safety and Tolerability of HM15321/GC1134A in Patients With Fabry Disease	RECRUITING	Fabry Disease	DRUG: HM15421/GC1134A	GC Biopharma Corp	INTERVENTIONAL
NCT07123155	2405N1221	RECRUITING	Pompe Disease
NCT06904261	AT1001-033	RECRUITING	Fabry Disease
NCT06121011	POM-005	RECRUITING	Pompe Disease
NCT06270316	CT-AMT-191-01	RECRUITING	Fabry Disease			INTERVENTIONAL
NCT07223944	FLT201-03	RECRUITING	Gaucher Disease		Freeline Therapeutics	INTERVENTIONAL
NCT06328608	CLI-06657AA1-01	RECRUITING	Fabry Disease
NCT07354724	DNLI-J-0001	RECRUITING	Pompe Disease
NCT05487599	A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)	ACTIVE NOT RECRUITING	Gaucher Disease| Gaucher Disease, Type 1	GENETIC: LY3884961	Prevail Therapeutics	INTERVENTIONAL
NCT04860960	Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1	ACTIVE NOT RECRUITING	Niemann-Pick Disease, Type C1	DRUG: Hydroxypropyl-beta-cyclodextrin| DRUG: Placebo	Cyclo Therapeutics, Inc.	INTERVENTIONAL
NCT02843035	Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension	ACTIVE NOT RECRUITING	Gaucher Disease Type 1| Gaucher Disease Type 3	DRUG: venglustat (GZ402671)|DRUG: imiglucerase	Genzyme, a Sanofi Company	INTERVENTIONAL
NCT06545136	FLT201-02	ACTIVE NOT RECRUITING	Gaucher Disease	GENETIC: FLT201	Freeline Therapeutics	INTERVENTIONAL
NCT05206773	A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease	ACTIVE NOT RECRUITING	Fabry Disease	DRUG: Venglustat (GZ402671)| DRUG: Placebo	Sanofi	INTERVENTIONAL
NCT06150820	AT845-02	ACTIVE NOT RECRUITING	Pompe Disease			INTERVENTIONAL
NCT05222906	Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3	ACTIVE NOT RECRUITING	Gaucher Disease Type 3	DRUG: Venglustat|DRUG: imiglucerase	Sanofi	INTERVENTIONAL
NCT05039866	ST-920-LT01	ACTIVE NOT RECRUITING	Fabry Disease	BIOLOGICAL: ST-920	Sangamo Therapeutics	INTERVENTIONAL
NCT05280548	A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease	ACTIVE NOT RECRUITING	Fabry Disease	DRUG: Venglustat (GZ402671) |DRUG: Agalsidase alfa |DRUG: Agalsidase beta (GZ419828) |DRUG: Migalastat	Sanofi	INTERVENTIONAL
NCT03737214	A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease	ACTIVE NOT RECRUITING	Fabry Disease	DRUG: Lucerastat	Idorsia Pharmaceuticals Ltd.	INTERVENTIONAL
NCT07640984	JR-446	NOT YET RECRUITING
NCT06109948	ABX1100-1001	CLOSED	Pompe Disease			INTERVENTIONAL
NCT02000310	13-CFCT-07	RECRUITING			Lysosomal and Rare Disorders Research and Treatment Center, Inc.	OBSERVATIONAL
NCT03812055	Cellular Pharmacodynamics of Small Molecules in Lysosomal Storage Disorders	RECRUITING	Lysosomal Storage Diseases		Lysosomal and Rare Disorders Research and Treatment Center, Inc.	OBSERVATIONAL
NCT05705674	The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3)	RECRUITING	Sanfilippo Disease		Lysosomal and Rare Disorders Research and Treatment Center, Inc.	OBSERVATIONAL
NCT06614894	24-LDRTC-01	RECRUITING			Lysosomal and Rare Disorders Research and Treatment Center, Inc.	OBSERVATIONAL
Not yet on CT.gov	FD24-LDRTC-03	RECRUITING	Fabry Disease		Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Not yet on CT.gov	GD25-LDRTC-01	RECRUITING	Gaucher Disease		Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Not yet on CT.gov	PD25-LDRTC-02	RECRUITING	Pompe Disease		Lysosomal and Rare Disorders Research and Treatment Center, Inc.
NCT06116071	23-LDRTC-02	ACTIVE NOT RECRUITING			Lysosomal and Rare Disorders Research and Treatment Center, Inc.	OBSERVATIONAL
NCT06065605	23-LDRTC-01	CLOSED			Lysosomal and Rare Disorders Research and Treatment Center, Inc.	OBSERVATIONAL