Status:
COMPLETED
Sapropterin in Individuals With Phenylketonuria
Lead Sponsor:
Washington University School of Medicine
Collaborating Sponsors:
BioMarin Pharmaceutical
University of Missouri-Columbia
Conditions:
Phenylketonuria
Eligibility:
All Genders
6-50 years
Brief Summary
Investigators at Washington University will examine the effects of sapropterin (Kuvan) on brain and cognition in individuals with phenylketonuria (PKU) using neuropsychological and neuroimaging proced...
Detailed Description
Investigators at Washington University will examine the effects of sapropterin (Kuvan) on brain and cognition in individuals with phenylketonuria (PKU) using neuropsychological and neuroimaging proced...
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 sapropoterin.
- For phenylketonuria,phenylalanine level greater than or equal to 450μmol/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 :
July 1 2008
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
May 1 2018
Estimated Enrollment :
45 Patients enrolled
Trial Details
Trial ID
NCT00730080
Start Date
July 1 2008
End Date
May 1 2018
Last Update
June 18 2018
Active Locations (1)
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1
Washington University
St Louis, Missouri, United States, 63130