NCT01082328

Response to Kuvan® in Subjects With Phenylketonuria (PKU) in a 4 Weeks Testing Period

ENDURE: A Phase IV, Prospective, Open-label, Uncontrolled, Multi-centre Cohort Trial to Assess the Responsiveness of Subjects With Phenylketonuria (PKU) to Treatment With Kuvan® 20 mg/kg/Day for 28 Days

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 59 (actual)
• Sponsor: Merck KGaA, Darmstadt, Germany · Industry
• Sex: All
• Age: 4 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of the study is to evaluate the proportion of responders (that is, greater than or equal to \[\>=\] 30 percent reduction from Baseline in blood phenylalanine \[Phe\] level) to treatment with Kuvan® (sapropterin dihydrochloride) 20 milligram per kilogram per day (mg/kg/day) for 28 days.

Conditions

• Phenylketonuria

Interventions

Type	Name	Description
DRUG	Kuvan®	Kuvan® (sapropterin dihydrochloride) oral solution 20 milligram per kilogram (mg/kg) will be given once daily for 28 +/- 1 days.

Timeline

• Start date: 2010-05-01
• Primary completion: 2012-05-01
• Completion: 2012-05-01
• First posted: 2010-03-08
• Last updated: 2014-02-27
• Results posted: 2013-08-01

Locations

1 site across 1 country: Norway