NCT00841100

Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 21 (actual)
• Sponsor: University of Miami · Academic / Other
• Sex: All
• Age: 4 Years
• Healthy volunteers: Not accepted

Summary

The investigators will investigate the effect of blood phenylalanine on Kuvan responsiveness in the same patients with PKU when their blood phenylalanine concentrations are altered by diet. Lowering blood phenylalanine concentrations in Kuvan non-responsive patients with PKU will increase the frequency of Kuvan responsiveness.

Conditions

• Phenylketonuria

Interventions

Type	Name	Description
DRUG	Kuvan	20mg/kg by mouth once daily
OTHER	Diet	Phenylalanine-restricted diet (4-10 mg/kg/day phenylalanine) using the prescribed phenylalanine-free medical formula and low protein foods tested with the patients for taste and acceptance to lower blood phenylalanine levels below 600 umol/l.

Timeline

• Start date: 2008-12-01
• Primary completion: 2011-07-19
• Completion: 2011-07-19
• First posted: 2009-02-11
• Last updated: 2019-12-03
• Results posted: 2019-10-08

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study