Clinical Trial: Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

Brief Summary: This is an open-label, non-comparative, Phase 3 study to evaluate the degree, frequency of response and safety of Kuvan® (sapropterin dihydrochloride) in subjects aged 4 to 18 years who have phenylketonuria and with elevated blood phenylalanine level of greater than or equal to 450 micromole per liter.

Detailed Summary:
Sponsor: Merck KGaA

Current Primary Outcome: Percentage of Participants With Response to Kuvan® (Sapropterin Dihydrochloride) Treatment [ Time Frame: Day 8 ]

Original Primary Outcome: 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 ]

Current Secondary Outcome:

• Percent Change From Baseline in Blood Phenylalanine Levels at Day 8 in Overall Population [ Time Frame: Baseline, Day 8 ]
  Percent change in blood phenylalanine levels after 8-day Kuvan® therapy (response test period) was calculated as (blood phenylalanine level at Day 8 minus blood phenylalanine level at baseline)*100/ blood phenylalanine level at baseline.
• Percent Change From Baseline in Blood Phenylalanine Levels at Day 8 in Sub-population of Responders [ Time Frame: Baseline, Day 8 ]
  Percent change in blood phenylalanine levels after 8-day Kuvan® therapy (response test period) was calculated as (blood phenylalanine level at Day 8 minus blood phenylalanine level at baseline)*100/ blood phenylalanine level at baseline.
• Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) in Overall Safety Population [ Time Frame: Baseline up to Week 11 ]
  An adverse event (AE) was defined as any new untoward medical occurrences/worsening of pre-existing medical condition without regard to possibility of causal relationship. A serious adverse event (SAE) was an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect.

Original Secondary Outcome:

• Percentage change from baseline in blood phenylalanine level after 8-day Kuvan® therapy [ Time Frame: Baseline and Day 8 ]
• Number of adverse events [ Time Frame: Baseline up to Week 11 (+/- 1 Week) ]

Dates:
Date Received: November 19, 2012
Date Started: November 2012
Date Completion:
Last Updated: July 25, 2014
Last Verified: July 2014