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

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Disease Information

Descriptive Information
Brief Title † A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302
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 Description 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.
Study Phase N/A
Study Type † Observational
Study Design †
Primary Outcome Measure † Changes in executive function, measured by CANTAB
Secondary Outcome Measure †
Condition † Phenylketonuria
Intervention † OtherAdministration of CANTAB and Subject Global Assessment
Study Arms / Comparison Groups
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Other
Estimated Enrollment † 100
Start Date † July 28, 2015
Completion Date April 2017
Primary Completion Date February 8, 2017
Eligibility Criteria † Inclusion Criteria: - Are currently participating in Part 1 of Study 165-302 and meet the criteria for participation in Part 2 of 165-302 - Are willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to any research-related procedures. - Have the ability to complete the CANTAB and subject global assessment. - Are willing and able to comply with all study procedures. Exclusion Criteria: - Any condition that, in the view of the investigator, places the subject at high risk of poor compliance or terminating early from the study
Gender All
Ages 18 Years - 70 Years
Accepts Healthy Volunteers No
Contacts ††
Location Countries † United States
Administrative Information
NCT ID † NCT02468570
Organization ID 165-303
Secondary IDs ††
Responsible Party Sponsor
Study Sponsor † BioMarin Pharmaceutical
Collaborators ††
Investigators † Study Director: Markus Merilainen, MD, Medical Director
Information Provided By
Verification Date January 2017
First Received Date † June 3, 2015
Last Updated Date January 30, 2017
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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