Phenylketonuria, Oxidative Stress, and BH4

• Status: Terminated
• Trial end date: 2013-06-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to see how tetrahydrobiopterin therapy (BH4; also known as sapropterin dihydrochloride or Kuvan) affects measures of oxidative stress and endothelial function in patients with Phenylketonuria (PKU).

• Phase: Phase 2
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: Emory University
• Treatments: Verapamil

Criteria

Inclusion Criteria:

• Have read, understood, and signed this consent form (and assent form, if <18 years old)

• Are between the ages of 10-45 years

• Weigh at least 75 pounds (34 kg)

• Meet group-specific criteria

Exclusion Criteria:

• Smoke

• Have any history of cardiovascular disease

• Have hepatic impairment, or demonstrate abnormal baseline liver function lab test results

• Have donated blood within 3 months prior to the study or are anemic as shown by the baseline lab results

• Are unwilling to discontinue dietary supplements (excluding medical food) two weeks prior to their first study visit

• Are currently take medications that will interfere with the interpretation of selected endpoints (such as lipid-lowering medication)

• Are currently pregnant or breastfeeding

• Excluding PKU, have a chronic illness, disorder, or condition (including but not limited to diabetes, hypertension, cancer)

• Have taken any investigational drug (including Kuvan for Kuvan-unresponsive participants & controls) within 3 months prior to the study

• In the opinion of the Investigator, should not be included in the study for any reason, including inability to follow study procedures