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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
Information provided by:
University of Miami Identifier:
First received: February 10, 2009
Last updated: March 10, 2009
Last verified: February 2009

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
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:

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


Ages Eligible for Study:   4 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00841100

Contact: Louis J Elsas, MD 305 243 7126
Contact: Andrea M Wierenga, PhD 305 243 8122

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

Additional Information:
Responsible Party: Dr. Louis J. Elsas, University of Miami 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:

Additional relevant MeSH terms:
Amino Acid Metabolism, Inborn Errors
Brain Diseases
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Central Nervous System Diseases
Genetic Diseases, Inborn
Metabolic Diseases
Metabolism, Inborn Errors
Nervous System Diseases processed this record on February 27, 2015