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Trial + Data / Events	Status	Ph	N	Region	Interventions	Sponsor	Conditions	Primary compl	Study compl
Bernat, John
LEAP2MONO, NCT05222906 / 2021-005402-10: Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3	Active, not recruiting	3	43	Europe, Canada, Japan, US, RoW	Venglustat, imiglucerase, Cerezyme®	Sanofi, Sanofi-aventis recherche & développement	Gaucher's Disease Type III	09/25	10/26
FLY, NCT06328608: A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease	Recruiting	2/3	22	Europe, US	PRX-102 1 mg/kg every two weeks, pegunigalsidase alfa, Recombinant human alpha galactosidase-A	Chiesi Farmaceutici S.p.A., ICON plc	Fabry Disease	12/27	03/28
NCT04145037: Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease	Terminated	1/2	8	Canada, US	AVR-RD-02	AVROBIO	Gaucher Disease	08/23	08/23
STAAR, NCT04046224 / 2019-000667-24: Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease	Active, not recruiting	1/2	34	Europe, Canada, US, RoW	ST-920	Sangamo Therapeutics	Fabry Disease	04/25	09/25
NCT06181136: Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)	Recruiting	1/2	20	US	DNL126	Denali Therapeutics Inc.	Mucopolysaccharidosis Type IIIA	08/28	08/28
NCT04552691: Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients	Approved for marketing	N/A		US	Pegunigalsidase Alfa	Chiesi Farmaceutici S.p.A., Chiesi USA, Inc.	Fabry Disease
NCT06488261: Long Term Follow-Up for Safety of AVR-RD-02	Active, not recruiting	N/A	2	US	AVR-RD-02	John Bernat	Gaucher Disease	06/37	10/37
Mistry, Pramod
LEAP2MONO, NCT05222906 / 2021-005402-10: Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3	Active, not recruiting	3	43	Europe, Canada, Japan, US, RoW	Venglustat, imiglucerase, Cerezyme®	Sanofi, Sanofi-aventis recherche & développement	Gaucher's Disease Type III	09/25	10/26
NCT05239858: International Wilson's Disease Patient Registry (iWilson Registry)	Recruiting	N/A	500	Europe, RoW		Orphalan, Ergomed	Wilson's Disease	12/27	12/27
Kopel, Teresa
LEAP2MONO, NCT05222906 / 2021-005402-10: Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3	Active, not recruiting	3	43	Europe, Canada, Japan, US, RoW	Venglustat, imiglucerase, Cerezyme®	Sanofi, Sanofi-aventis recherche & développement	Gaucher's Disease Type III	09/25	10/26
STAAR, NCT04046224 / 2019-000667-24: Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease	Active, not recruiting	1/2	34	Europe, Canada, US, RoW	ST-920	Sangamo Therapeutics	Fabry Disease	04/25	09/25