A Study of CNSA-001 in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
Status:
Completed
Trial end date:
2021-01-09
Target enrollment:
Participant gender:
Summary
This study has been designed to demonstrate the safety, pharmacokinetics (PK) and preliminary
efficacy of CNSA-001 in reducing blood phenylalanine concentrations in participants with
hyperphenylalaninemia due to primary tetrahydrobiopterin (BH4) deficiency.