Urinary phenylacetylglutamine as dosing biomarker for patients with urea cycle disorders.

ABSTRACT:

Unlabelled

We have analyzed pharmacokinetic data for glycerol phenylbutyrate (also GT4P or HPN-100) and sodium phenylbutyrate with respect to possible dosing biomarkers in patients with urea cycle disorders (UCD).

Study design

These analyses are based on over 3000 urine and plasma data points from 54 adult and 11 pediatric UCD patients (ages 6-17) who participated in three clinical studies comparing ammonia control and pharmacokinetics during steady state treatment with glycerol phenylbutyrate or sodium phenylbutyrate. All patients received phenylbutyric acid equivalent doses of glycerol phenylbutyrate or sodium phenylbutyrate in a cross over fashion and underwent 24-hour blood samples and urine sampling for phenylbutyric acid, phenylacetic acid and phenylacetylglutamine.

SUBMITTER: Mokhtarani M 

PROVIDER: S-EPMC3608516 | biostudies-literature | 2012 Nov

REPOSITORIES: biostudies-literature