The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)

• Status: Completed
• Trial end date: 2011-08-01
• Target enrollment: 0
• Participant gender: All

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.

• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: University of Missouri-Columbia
• Collaborator: BioMarin Pharmaceutical
• Treatments: Verapamil

Criteria

Inclusion Criteria:

• 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 Criteria:

• 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.