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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:
University of Miami

February 10, 2009
February 11, 2009
March 12, 2009
December 2008
February 2010   (Final data collection date for primary outcome measure)
Evaluation of effect of blood phenylalanine on Kuvan response [ Time Frame: 18 months ]
Same as current
Complete list of historical versions of study NCT00841100 on ClinicalTrials.gov Archive Site
Not Provided
Not Provided
Not Provided
Not Provided
 
Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response
Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response

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.

Not Provided
Interventional
Not Applicable
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Phenylketonuria
Drug: Kuvan
20mg/kg/d, once daily
Other Name: sapropterin dihydrochloride (tetrahydrobiopterin, 6R-BH4
Experimental: Kuvan and nutrition couseling
Intervention: Drug: Kuvan
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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Unknown status
20
Same as current
February 2010
February 2010   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
4 Years and older   (Child, Adult, Older Adult)
No
Contact information is only displayed when the study is recruiting subjects
United States
 
 
NCT00841100
20080675
Yes
Not Provided
Not Provided
Dr. Louis J. Elsas, University of Miami
University of Miami
Not Provided
Principal Investigator: Louis J Elsas, MD University of Miami
University of Miami
February 2009

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP