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Trial record 18 of 53 for:    "Phenylketonuria"
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Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response

The recruitment status of this study is unknown because the information has not been verified recently.
Verified February 2009 by University of Miami.
Recruitment status was  Recruiting
Sponsor:
Information provided by:
University of Miami
ClinicalTrials.gov Identifier:
NCT00841100
First received: February 10, 2009
Last updated: March 10, 2009
Last verified: February 2009
History of Changes
  Purpose

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 Intervention
Phenylketonuria
 Drug: Kuvan

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response

Resource links provided by NLM:

MedlinePlus related topics: Phenylketonuria
U.S. FDA Resources

Further study details as provided by University of Miami:

Primary Outcome Measures:
  • Evaluation of effect of blood phenylalanine on Kuvan response [ Time Frame: 18 months ] [ Designated as safety issue: No ]

Estimated Enrollment: 20
Study Start Date: December 2008
Estimated Study Completion Date: February 2010
Estimated Primary Completion Date: February 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Kuvan and nutrition couseling Drug: Kuvan
20mg/kg/d, once daily
Other Name: sapropterin dihydrochloride (tetrahydrobiopterin, 6R-BH4

  Eligibility

Ages Eligible for Study:   4 Years and older
Genders Eligible for Study:   Both
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
Please refer to this study by its ClinicalTrials.gov identifier: 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:
Website about tetrahydrobiopterin  This link exits the ClinicalTrials.gov site

Publications:

Responsible Party: Dr. Louis J. Elsas, University of Miami
ClinicalTrials.gov Identifier: NCT00841100     History of Changes
Other Study ID Numbers: 20080675
Study First Received: February 10, 2009
Last Updated: March 10, 2009
Health Authority: United States: Institutional Review Board

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

ClinicalTrials.gov processed this record on June 18, 2013