Behavioral Effects of Kuvan in Children With Mild Phenylketonuria

• Status: Terminated
• Trial end date: 2010-01-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to determine whether improvements in behavior occur in children with phenylketonuria (PKU) who are taking Kuvan.

• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Washington University School of Medicine
• Collaborators: BioMarin Pharmaceutical; Northwestern University; Oregon Health and Science University; University of Missouri-Columbia

Criteria

Inclusion Criteria:

• 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 Criteria:

• 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.