Active	Status	Sponsor type	Region	Phase	Filter
5 Active 7 Inactive 12 All	Not yet recruiting Recruiting Enrolling by invitation Active, not recruiting Available No longer recruiting	Industry lead sponsor Industry collaborator Coop Other	US Canada Japan Europe RoW	N/A 1 2 3 4	Highlight changes:- Show only changed items Enrollment:

Advanced Filters

4 Trials

Active Trials   Industry lead sponsorIndustry collaborator

 

Export

Trial + Data / Events	Status	Ph	N	Region	Interventions	Sponsor	Conditions	Primary compl	Study compl
CHIEN, Yin-Hsiu
NCT02926066 / 2019-003072-39: A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion	Completed	2	12	RoW	AAV2-hAADC	National Taiwan University Hospital, PTC Therapeutics	Aromatic Amino Acid Decarboxylase Deficiency	01/22	05/22
NCT04903288: A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants	Active, not recruiting	2	13	US, RoW	Eladocagene Exuparvovec, KEBILIDI™	PTC Therapeutics	AADC Deficiency	05/23	04/28
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
NCT04174157: Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)	Recruiting	N/A	700	Europe, Japan, US, RoW	Prospective observational registry, Zolgensma	Novartis Pharmaceuticals, United BioSource, LLC	Spinal Muscular Atrophy (SMA)	06/38	06/38