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Trial + Data / Events	Status	Ph	N	Region	Interventions	Sponsor	Conditions	Primary compl	Study compl
McNutt, Markey
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
NCT05099640 / 2021-000474-29: A Study of PTC923 in Participants With Phenylketonuria	Completed	3	157	Europe, Canada, US, RoW	PTC923, Placebo	PTC Therapeutics	Phenylketonuria	04/23	05/23
SYNPHENY-3, NCT05764239: Efficacy and Safety of SYNB1934 in Patients With PKU	Terminated	3	35	Canada, US, RoW	SYNB1934v1, Placebo	Synlogic	Phenylketonuria	03/24	03/24
NCT05166161 / 2021-000497-28: A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria	Recruiting	3	200	Europe, Canada, Japan, US, RoW	PTC923, Sepiapterin	PTC Therapeutics	Phenylketonuria	04/26	04/26
CACN00177-100D, NCT05154890 / 2019-004791-19: A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency	Terminated	1/2	13	Europe, US, RoW	Pegtarviliase IV, ACN00177, Pegtarviliase SC	Aeglea Biotherapeutics, Aeglea Biotherapeutics	Homocystinuria Due to Cystathionine Beta-Synthase Deficiency	04/23	04/23
NCT06332807: AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)	Recruiting	1/2	12	US	NGGT002	NGGT INC.	Phenylketonurias	12/30	12/30
NCT05222178: Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency	Terminated	1	3	US	HMI-103	Homology Medicines, Inc	Phenylketonurias, PAH Deficiency, Phenylketonuria	09/23	09/23