Status:
COMPLETED
The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)
Lead Sponsor:
University of Missouri-Columbia
Collaborating Sponsors:
BioMarin Pharmaceutical
Conditions:
Phenylketonuria
Eligibility:
All Genders
6+ years
Brief Summary
The objective of the study is to determine whether Kuvan™ (sapropterin) improves the strength of the functional connectivity between brain regions in individuals with PKU.
Detailed Description
The proposed study is designed to evaluate the hypothesis that functional connectivity may represent an early marker for neurocognitive improvements related to Kuvan treatment. As a first step in the ...
Eligibility Criteria
Inclusion
- Willing and able to provide informed consent or assent.
- Willing and able to comply with study procedures.
- Greater than or equal to 6 years of age.
- For phenylketonuria, intention of physician to prescribe sapropterin.
- For phenylketonuria, phenylalanine level greater than or equal to 300μmol/L.
- For phenylketonuria, negative pregnancy test if of childbearing potential.
- For phenylketonuria, willing to use contraception if sexually active.
Exclusion
- 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.
- Perceived to be unreliable or unavailable for study.
- Use of L-Dopa, methotrexate, or other drugs that inhibit folate metabolism.
- For phenylketonuria, known hypersensitivity to sapropterin or excipients.
Key Trial Info
Start Date :
August 1 2009
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
August 1 2011
Estimated Enrollment :
20 Patients enrolled
Trial Details
Trial ID
NCT00964236
Start Date
August 1 2009
End Date
August 1 2011
Last Update
January 4 2018
Active Locations (1)
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1
University of Missouri
Columbia, Missouri, United States, 65211