Long-Term Tetrahydrobiopterin Treatment in PKU Patients of 0-18 Years - Study on Phenylalanine Tolerance and Safety
Status:
Terminated
Trial end date:
1969-12-31
Target enrollment:
Participant gender:
Summary
The aim of the study is to confirm the efficacy and safety of BH4 in the treatment of
hyperphenylalaninemia caused by phenylalanine hydroxylase deficiency in patients responsive
to BH4. The primary objective is to assess the effect of BH4 on phenylalanine tolerance
compared to placebo under optimal blood phenylalanine control and to demonstrate safety in 12
months long-term treatment. Additionally population PK will be assessed.