Status:
TERMINATED
Behavioral Effects of Kuvan in Children With Mild Phenylketonuria
Lead Sponsor:
Washington University School of Medicine
Collaborating Sponsors:
BioMarin Pharmaceutical
University of Missouri-Columbia
Conditions:
Phenylketonuria
Eligibility:
All Genders
6-18 years
Brief Summary
The purpose of this study is to determine whether improvements in behavior occur in children with phenylketonuria (PKU) who are taking Kuvan.
Detailed Description
Little research has been conducted to examine behavior and cognition in children with mild PKU/hyperphenylalanemia, but there is evidence of reductions in general intelligence (IQ) (Costello, 1994) an...
Eligibility Criteria
Inclusion
- Willing and able to provide informed consent and/or assent.
- Willing and able to comply with study procedures.
- Between 6 and 18 years of age, inclusive.
- Intention of physician to prescribe Kuvan.
- Phenylalanine levels between 360μmol/L and 600μmol/L, inclusive, when untreated with dietary restrictions.
- Negative pregnancy test if of childbearing potential.
- Willing to use contraception if sexually active.
Exclusion
- Treatment with Kuvan within the past 6 months.
- Pregnant, breastfeeding, or planning to become pregnant during study.
- Use of investigational product less than 30 days prior to or during study.
- Concurrent condition that could interfere with participation or safety.
- Any condition creating high risk of poor compliance with study.
- History of major medical disorder unrelated to phenylketonuria.
- Perceived to be unreliable or unavailable for study.
- Use of L-Dopa, methotrexate, or other drugs that inhibit folate metabolism.
- Known hypersensitivity to sapropterin or excipients.
Key Trial Info
Start Date :
January 1 2009
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
January 1 2010
Estimated Enrollment :
2 Patients enrolled
Trial Details
Trial ID
NCT00827762
Start Date
January 1 2009
End Date
January 1 2010
Last Update
December 5 2013
Active Locations (4)
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1
Northwestern University/Children's Memorial Hospital
Chicago, Illinois, United States, 60614
2
University of Missouri
Columbia, Missouri, United States, 65211
3
Washington University
St Louis, Missouri, United States, 63130
4
Oregon Health & Science University
Portland, Oregon, United States, 97239