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

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Disease Information

Descriptive Information
Brief Title † Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response
Official Title † Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response
Brief 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.
Detailed Description
Study Phase N/A
Study Type † Interventional
Study Design † Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary Outcome Measure † Evaluation of effect of blood phenylalanine on Kuvan response
Secondary Outcome Measure †
Condition † Phenylketonuria
Intervention † DrugKuvan
Study Arms / Comparison Groups Kuvan and nutrition couseling
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Drug
Estimated Enrollment † 20
Start Date † December 2008
Completion Date February 2010
Primary Completion Date February 2010
Eligibility Criteria † 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
Gender Both
Ages 4 Years - N/A
Accepts Healthy Volunteers No
Contacts †† Louis J Elsas, MD, 305 243 7126, lelsas@med.miami.edu
Location Countries † United States
Administrative Information
NCT ID † NCT00841100
Organization ID 20080675
Secondary IDs ††
Responsible Party
Study Sponsor † University of Miami
Collaborators ††
Investigators † Principal Investigator: Louis J Elsas, MD, University of Miami
Information Provided By
Verification Date February 2009
First Received Date † February 10, 2009
Last Updated Date March 10, 2009
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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