Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Patients With Phenylketonuria
This is an open-label, non-comparative, Phase 3 study to evaluate the degree, frequency of response and safety of Kuvan® in subjects aged 4 to 18 years who have phenylketonuria with elevated blood phenylalanine level of greater than or equal to 450 micromole per liter.
|Study Design:||Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase III Non-comparative Open-label Clinical Study to Evaluate the Response to and Safety of Kuvan® (Sapropterin Dihydrochloride) After 6 Weeks of Treatment in Patients of 4 to 18 Years of Age With Phenylketonuria Who Have Elevated Blood Phenylalanine Levels|
- Response to Kuvan® after 8-day treatment defined as a reduction in blood Phenylalanine levels of greater than or equal to 30 percent as compared to beginning of the test prior to dosing [ Time Frame: Day 8 ] [ Designated as safety issue: No ]
- Percentage change from baseline in blood phenylalanine level after 8-day Kuvan® therapy [ Time Frame: Baseline and Day 8 ] [ Designated as safety issue: No ]
- Number of adverse events [ Time Frame: Baseline up to Week 11 (+/- 1 Week) ] [ Designated as safety issue: Yes ]
|Study Start Date:||November 2012|
|Study Completion Date:||October 2013|
|Primary Completion Date:||October 2013 (Final data collection date for primary outcome measure)|
Kuvan® oral soluble tablet will be administered once daily in the morning, as 20 milligram per kilogram of the subject's body mass. The therapy duration will depend on the response to treatment - either up to 8 days, (if there will be no response), or up to 7 weeks (if there will be response).