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Lexington, Kentucky 40536

  • Phenylketonuria

Purpose:

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


Study 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.


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


Study is Available At:


Original ID:

165-303


NCT ID:

NCT02468570


Secondary ID:


Study Acronym:

PRISM303


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


Overall Status:

Completed


Study Phase:

N/A


Genders:

N/A


Minimum Age:

18 Years


Maximum Age:

70 Years


Quick Facts

Healthy Volunteers
Oversight Has DMC
Study Is FDA Regulated
Study Is Section 801
Has Expanded Access

Study Source:

BioMarin Pharmaceutical


Oversight Authority:

United States: Food and Drug Administration


Reasons Why Stopped:


Study Type:

Observational


Study Design:


Number of Arms:

0


Number of Groups:

1


Total Enrollment:

9


Enrollment Type:

Actual


Overall Contact Information

Official Name:Debra Lounsbury
Study Director
Principal Scientist/Medical Monitor

Study Dates

Start Date:July 28, 2015
Completion Date:February 7, 2017
Completion Type:Actual
Primary Completion Date:February 7, 2017
Primary Completion Type:Actual
Verification Date:December 2017
Last Changed Date:December 7, 2017
First Received Date:June 3, 2015

Study Outcomes

Outcome Type:Primary Outcome
Measure:Changes in executive function, measured by CANTAB
Time Frame:63 weeks
Safety Issues:False
Description:will be assessed by using a selected set of three tasks

Study Interventions

Intervention Type:Other
Name:Administration of CANTAB and Subject Global Assess
Description:At designated visits, subject will perform the CANTAB tasks and complete the Subject Global Assessment Questionnaire

Study Arms

There are no available Study Arms

Study Agencies

Agency Class:Industry
Agency Type:Lead Sponsor
Agency Name:BioMarin Pharmaceutical

Samples and Retentions

Study Population: Subjects with PKU who are currently enrolled in 165-302 Part 2.
Sample Method:Non-Probability Sample

Study References

There are no available Study References

Data Source: ClinicalTrials.gov

Date Processed: March 30, 2020

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