NCT00272792

Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet

A Phase 3, Double-blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 45 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 4 Years – 12 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this trial is to evaluate the ability of Phenoptin to increase phenylalanine (phe) tolerance in children with phenylketonuria who are following a phe-restricted diet.

Conditions

• Phenylketonurias

Interventions

Type	Name	Description
DRUG	Sapropterin Dihydrochloride	Phenoptin, provided in tablets containing 100 mg of sapropterin dihydrochloride each, was administered orally once daily in the morning as the number of tablets equivalent to a 20mg/kg/day dose dissolved in 4-8 oz (120-240 mL) of water or apple juice for 6 weeks. A follow-up call or visit was made 4 weeks later during this double-blind, placebo-controlled study.
DRUG	Placebo	Placebo, provided as tablets similar to Phenoptin tablets, was administered orally once daily in the morning as the number of tablets equivalent to a 20mg/kg/day dose dissolved in 4 8 oz (120-240 mL) of water or apple juice. for 6 weeks. A follow-up call or visit was made 4 weeks later during this double-blind, placebo-controlled study.

Timeline

• Start date: 2006-02-01
• Completion: 2006-11-01
• First posted: 2006-01-09
• Last updated: 2015-08-13
• Results posted: 2009-07-09

Locations

9 sites across 1 country: United States