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

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ClinicalTrials.gov Identifier: NCT00841100
Recruitment Status : Unknown
Verified February 2009 by University of Miami.
Recruitment status was:  Recruiting
First Posted : February 11, 2009
Last Update Posted : March 12, 2009
Sponsor:
Information provided by:

Study Description
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.


Condition or disease Intervention/treatment
Phenylketonuria Drug: Kuvan

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response
Study Start Date : December 2008
Estimated Primary Completion Date : February 2010
Estimated Study Completion Date : February 2010


Arms and Interventions

Arm Intervention/treatment
Experimental: Kuvan and nutrition couseling Drug: Kuvan
20mg/kg/d, once daily
Other Name: sapropterin dihydrochloride (tetrahydrobiopterin, 6R-BH4


Outcome Measures

Primary Outcome Measures :
  1. Evaluation of effect of blood phenylalanine on Kuvan response [ Time Frame: 18 months ]

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00841100


Contacts
Contact: Louis J Elsas, MD 305 243 7126 lelsas@med.miami.edu
Contact: Andrea M Wierenga, PhD 305 243 8122 awierenga@med.miami.edu

Locations
United States, Florida
Department of Biochemistry & Molecular Biology Recruiting
Miami, Florida, United States, 33136
Contact: Louis J Elsas, MD    305-243-7126    lelsas@med.miami.edu   
Contact: Andrea M Wierenga, PhD    305 243 8122    awierenga@med.miami.edu   
Sub-Investigator: Andrea M Wierenga, PhD         
Sponsors and Collaborators
University of Miami
Investigators
Principal Investigator: Louis J Elsas, MD University of Miami
More Information

Additional Information:
Publications:
Responsible Party: Dr. Louis J. Elsas, University of Miami
ClinicalTrials.gov Identifier: NCT00841100     History of Changes
Other Study ID Numbers: 20080675
First Posted: February 11, 2009    Key Record Dates
Last Update Posted: March 12, 2009
Last Verified: February 2009

Keywords provided by University of Miami:
PKU
Kuvan
BH4
tetrahydrobiopterin

Additional relevant MeSH terms:
Phenylketonurias
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases