Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria
Status:
Completed
Trial end date:
2005-11-01
Target enrollment:
Participant gender:
Summary
The primary objective is to evaluate the degree and frequency of response to Phenoptin™
(sapropterin dihydrochloride), as demonstrated by a reduction in blood phenylalanine (Phe)
level among subjects with phenylketonuria (PKU) who have elevated Phe levels. A secondary
objective of this study is to evaluate the safety of Phenoptin™ treatment in this subject
population, and identify individuals in this subject population who respond to Phenoptin™
treatment with a reduction in blood Phe level.