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

Completed · Phase 2

Conditions studied: Phenylketonuria

In brief

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.

Key facts

Study ID

NCT00841100

Run by

University of Miami

People needed

21

Starts

2008-12-01

Expected to finish

2011-07-19

Last updated by the study team

2019-12-03

Who can join

Age: 4 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• all patients with a diagnosis of phenylketonuria
• patients who are over the age of 4 years,
• patients with a fasting blood phenylalanine ≥600µmol/l

You may not qualify if…

• 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

Where it is running

• Department of Biochemistry & Molecular Biology — Miami, Florida, United States

Full record on ClinicalTrials.gov

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