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

Phenylketonuria Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00841100
• Other study ID #: 20080675
• Status: Completed
• Phase: Phase 2
• Start date: December 2008
• Est. completion date: July 19, 2011

Study information

• Verified date: November 2019
• Source: University of Miami
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 21
• Est. completion date: July 19, 2011
• Est. primary completion date: July 19, 2011
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years and older

Eligibility

Inclusion Criteria:

• all patients with a diagnosis of phenylketonuria

• patients who are over the age of 4 years,

• patients with a fasting blood phenylalanine =600µmol/l

Exclusion Criteria:

• female patients who are pregnant or attempting to become pregnant

• children under four years of age

• Concurrent disease of condition that would interfere with study participation or safety such as history of neurological compromise or major medical, psychiatric or learning disorder unrelated to PKU

• Any condition that, in the view of the Investigators, places subject at high risk of poor treatment compliance or of not completing the study

• Use of L-Dopa, methotrexate, or other drugs that may inhibit folate metabolism

Related Conditions & MeSH terms

• Phenylketonuria
• Phenylketonurias

Intervention

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.

Locations

Country	Name	City	State
United States	Department of Biochemistry & Molecular Biology	Miami	Florida

Sponsors (1)

Lead Sponsor	Collaborator
University of Miami

Country where clinical trial is conducted

United States, 

References & Publications (1)

Burton BK, Grange DK, Milanowski A, Vockley G, Feillet F, Crombez EA, Abadie V, Harding CO, Cederbaum S, Dobbelaere D, Smith A, Dorenbaum A. The response of patients with phenylketonuria and elevated serum phenylalanine to treatment with oral sapropterin dihydrochloride (6R-tetrahydrobiopterin): a phase II, multicentre, open-label, screening study. J Inherit Metab Dis. 2007 Oct;30(5):700-7. Epub 2007 Sep 12. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percent Change in Blood Phenylalanine	Percent change in phenylalanine in (uM) on Kuvan Response evaluated via fasting morning blood serum. A decrease of 30% or greater indicates positive response on Kuvan.	Baseline to Day 1 of the Acute Phase, Baseline to Day 28 of Phase 1, Baseline to Day 28 of Phase 3