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 [ Designated as safety issue: No ]
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 symptoms [ Designated as safety issue: No ]Compare 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 biomarkers [ Designated as safety issue: No ]Explore 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|