NCT00104260

Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria

A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 700 (planned)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 8 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Phenylketonurias

Interventions

Type	Name	Description
DRUG	sapropterin dihydrochloride

Timeline

• Start date: 2004-12-01
• Completion: 2005-11-01
• First posted: 2005-02-25
• Last updated: 2007-04-09

Locations

13 sites across 1 country: United States