NCT01732471

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

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 90 (actual)
• Sponsor: Merck KGaA, Darmstadt, Germany · Industry
• Sex: All
• Age: 4 Years – 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Phenylketonuria

Interventions

Type	Name	Description
DRUG	Kuvan®	Kuvan® (sapropterin dihydrochloride) will be administered orally at a dose of 20 milligram per kilogram per day (mg/kg/day) once daily for 8 days. If there is 30 percent (%) decrease in blood phenylalanine levels from baseline at the end of Day 8, then treatment will be continued at the same dose for further 6 weeks.

Timeline

• Start date: 2012-11-01
• Primary completion: 2013-10-01
• Completion: 2013-10-01
• First posted: 2012-11-22
• Last updated: 2014-07-28
• Results posted: 2014-07-28

Locations

1 site across 1 country: Germany