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Trial + Data / Events	Status	Ph	N	Region	Interventions	Sponsor	Conditions	Primary compl	Study compl
Longo, Nicola
NCT03335488 / 2015-000075-27: Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)	Completed	4	16	Europe, US	RAVICTI, Glycerol phenylbutyrate, GPB, HPN-100, NaPBA, Sodium phenylbutyrate	Amgen	Urea Cycle Disorder	07/22	12/22
PERIDOT, NCT05206773 / 2021-002350-90: 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	3	122	Europe, Canada, Japan, US, RoW	Venglustat (GZ402671), Placebo	Sanofi, Sanofi-Aventis Recherche & Développement	Fabry Disease	12/25	12/26
FORTIS, NCT04174105: Gene Transfer Study in Patients With Late Onset Pompe Disease	Recruiting	1/2	18	Europe, US	zocaglusagene nuzaparvovec, AT845	Astellas Gene Therapies	Pompe Disease (Late-onset)	11/29	11/29
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
Wever-Pinzon, Omar
DEFINE-HT, NCT05309382: Development of Non-invasive Cell-free DNA to Supplant Invasive Biopsy in Heart Transplantation	Completed	N/A	147	US	Prospera	Natera, Inc.	Heart Transplant	10/24	10/24