The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical PKU
This study is an independent sub-study of the protocol titled PKU-016: A double-blind, placebo-controlled, randomized study to evaluate the safety and therapeutic effects of sapropterin dihydrochloride on neuro-psychiatric symptoms in subjects with phenylketonuria (PKU ASCEND).
The primary objective of this study is to determine oxidative stress in patients with classical phenylketonuria (PKU) enrolled in PKU-016, using a brain scan (called an HMPAO SPECT) at baseline and 26 weeks, and blood redox biomarkers.
|Study Design:||Time Perspective: Prospective|
|Official Title:||The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical Phenylketonuria|
- Determine in vivo redox status in patients with classical phenylketonuria
The primary objective of this study is to determine in vivo redox status in patients with classical phenylketonuria enrolled in PKU-016, compared to historical normal controls, using:
- Serial Tc99m-HMPAO SPECT brain imaging (baseline and 26 weeks); and
- Blood redox biomarkers, including oxidized and reduced glutathione, tetrahydrobiopterin, ascorbate, alpha-tocotrienol, selenium, etc.
- Compare redox status with neuropsychological and neuro-cognitive symptomsCompare redox status as determined by brain imaging and blood redox biomarkers to measures of neuropsychological and neuro-cognitive symptoms (ADHD, anxiety, depression and executive function) and global function using data collected as part of PKU-016
- Explore the utility of other blood redox biomarkersExplore the utility of other blood redox biomarkers (e.g. NAD+/NADH, NADP+/NADPH, protein carbonyls) in determining level of oxidative stress.
Biospecimen Retention: Samples With DNA
|Study Start Date:||July 2012|
Please refer to this study by its ClinicalTrials.gov identifier: NCT01650909
|Principal Investigator:||Gregory Enns, MD||Stanford University|