The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical PKU

Classical Phenylketonuria(PKU) Clinical Trial

Official title: The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical Phenylketonuria

Status Withdrawn

Clinical Trial Summary

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.

Clinical Trial Description

n/a

Study Design

Time Perspective: Prospective

Related Conditions & MeSH terms

• Classical Phenylketonuria(PKU)
• Phenylketonurias

• NCT number: NCT01650909
• Study type: Observational
• Source: Stanford University
• Status: Withdrawn
• Phase: Phase 3
• Start date: July 2012