Phenylketonuria, Oxidative Stress, and BH4

Stopped early · Phase 2

Conditions studied: Phenylketonuria

In brief

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).

Key facts

Study ID: NCT01395394
Run by: Emory University
People needed: 12
Starts: 2011-06-01
Expected to finish: 2013-06-01
Last updated by the study team: 2014-08-28

Who can join

Age: 10 and older, up to 45. Sex: any. Healthy volunteers: accepted.

You may qualify if…

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

You may not qualify if…

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

Where it is running

Emory University — Decatur, Georgia, United States

Full record on ClinicalTrials.gov

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