Clinical Trial: A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Observational

Official Title: A Phase 3 Substudy to Evaluate Executive Function in Adults With Phenylketonuria Who Are Participating in the Phase 3 Study, 165-302

Brief Summary: Phase 3 substudy enrolling approximately 100 subjects, aged ≥ 18 to ≤ 70 years old, with PKU who are concurrently participating in the 165-302 study.

Detailed Summary: This is a Phase 3 substudy enrolling approximately 100 subjects, aged ≥ 18 to ≤ 70 years old, with PKU who are concurrently treated with BMN 165 or placebo in Part 2 and BMN 165 in Part 4 of Study 165-302. No study drug is administered as part of this study. For this study, subjects will be asked to perform computer-based assessments (CANTAB) that assess executive function (specifically, attention, working memory, cognitive flexibility) and to answer questions about their current state of self perception (subject global assessment comprised of seven questions). The study visits are as follows: screening, baseline visit, and 3 additional study visits at different time points in the study.
Sponsor: BioMarin Pharmaceutical

Current Primary Outcome: Changes in executive function, measured by CANTAB [ Time Frame: 63 weeks ]

will be assessed by using a selected set of three tasks


Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: BioMarin Pharmaceutical

Dates:
Date Received: June 3, 2015
Date Started: July 28, 2015
Date Completion: April 2017
Last Updated: January 30, 2017
Last Verified: January 2017