Active	Status	Sponsor type	Region	Phase	Filter
7 Active 2 Inactive 9 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:

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7 Trials

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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
Billy, Jenny
PEGASUS, NCT05270837 / 2021-005058-27: Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria	Active, not recruiting	3	55	Europe, US	Pegvaliase, PEG PAL, BMN 165, Diet Only	BioMarin Pharmaceutical, BioMarin Pharmaceutical Inc.	Phenylketonuria (PKU)	01/25	10/27
Fait, Kenzie
HERO, NCT04732429 / 2023-000785-33: Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia	Terminated	2	26	US, RoW	HST5040, Placebo	HemoShear Therapeutics, HemoShear Therapeutics	Methylmalonic Acidemia, Propionic Acidemia	10/23	10/23
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