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BH4 Deficiency clinical trials

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NCT ID: NCT03519711 Completed - Clinical trials for Hyperphenylalaninemia

A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia

Start date: January 3, 2019
Phase: Phase 1/Phase 2
Study type: Interventional

This study has been designed to demonstrate the safety, pharmacokinetics (PK) and preliminary efficacy of PTC923 (CNSA-001) in reducing blood phenylalanine concentrations in participants with hyperphenylalaninemia due to primary BH4 deficiency (PBD).