Response to Phenylketonuria to Tetrahydrobiopterin (BH4)

• Status: Terminated
• Trial end date: 2015-01-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to determine whether tetrahydrobiopterin (BH4)is effective in treating patients with PKU.

• Phase: Phase 1
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: FDA Office of Orphan Products Development; The University of Texas Medical Branch, Galveston

Criteria

Inclusion Criteria:

• Subject and/or parent or guardian must be capable of understanding and providing written informed consent

• Subjects must have Phenylketonuria (PKU)or hyperphenylalaninemia (HPA), defined as baseline blood Phe levels of >600 umol/L

• Subjects must be at least 10 years of age, and may be of either gender and any ethnic group

• Female subjects of childbearing potential must agree to use adequate birth control or refrain from sexual activity throughout study participation

Exclusion Criteria:

• Female subjects who are pregnant or breastfeeding

• Subjects who have concurrent diseases or conditions that require medication or treatment

• Subjects who require concomitant treatment with any drug known to inhibit folate synthesis

• Subjects who have been treated with any investigational drug within 30 days