CNSA-001 ( DrugBank: - )


1 disease
IDDisease name (Link within this page)Number of trials
240Phenylketonuria1

240. Phenylketonuria


Clinical trials : 138 Drugs : 106 - (DrugBank : 11) / Drug target gene : 1 - Drug target pathways : 5
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1NCT03519711
(ClinicalTrials.gov)
June 24, 201811/4/2018A Study of CNSA-001 in Primary Tetrahydrobiopterin (BH4) Deficient Participants With HyperphenylalaninemiaA Phase 1/2, Open-Label, Randomized Parallel Arm, Intra-patient Dose Escalation Study to Evaluate the Safety, Pharmacokinetics and Preliminary Efficacy of CNSA-001(Sepiapterin) in Primary Tetrahydrobiopterin Deficient Patients With HyperphenylalaninemiaBH4 Deficiency;HyperphenylalaninemiaDrug: CNSA-001PTC TherapeuticsNULLCompleted12 MonthsN/AAll6Phase 1/Phase 2United States;Germany