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

NCT02468570 | Completed Results

• 1 other identifier: 165-303
• Study Type: observational
• Target: 9
• Locations: 1 country
• Sites: 4

Brief Summary

A Phase 3 substudy to evaluate executive function in adults with phenylketonuria who are participating in the phase 3 Study, 165-302. Approximately 100 subjects, aged ≥ 18 to ≤ 70 years old, with PKU will be enrolled.

Conditions

Phenylketonuria

Keywords

PKU; PEG-PAL; Pegvaliase; BMN 165; rAv-PAL PEG; PRISM 302; PRISM 303; BioMarin; 165-302

Outcome Measures

Primary Outcomes (3)

• Change From Baseline in Rapid Visual Processing (RVP) Mean Response Latency (CANTAB Task)

  The RVP task measures sustained attention. In this task, a series of random digits continuously appears in the middle of the screen. Subjects are asked to recognize a specific sequence of digits and are asked to press a button on the screen whenever this specific sequence of digits appears within this series of continuous digits. RVP mean latency is the mean response latency during assessment sequence blocks where the subject responded correctly (Latency units are milliseconds). Lower scores represent better performance. Observed range for Pooled Active: -103.1 to 65.5 (change from baseline) Observed range for Pooled Placebo: -10.4 to 39.0 (change from baseline)

  Baseline to Part 2 Week 8

• Change From Baseline in Spatial Working Memory (SWM) Between Errors 4-8 Boxes (CANTAB Task)

  The SWM task measures visuospatial working memory. During this task, a number of boxes appears on a screen. The objective of the task is to find a token under a box and to place this token in a designated area. There is only one token under one box during each round and the location of the token rotates in each round to a different box, other than a box within which the token was found in previous rounds. The subject is therefore asked to remember which boxes previously contained tokens so as to better the chances and speed of finding tokens under boxes which did not previously contain tokens. SWM between errors measures the total number of times the subject revisits a box in which a token has previously been found in the same problem (calculated for assessed problems only). Observed range for Pooled Active: -8 to 1 (change from baseline) Observed range for Pooled Placebo: -5 to 12 (change from baseline) Lower scores represent better performance.

  Baseline to Part 2 Week 8

• Change From Baseline in Stop Signal Task (SST) Stop Signal Reaction T (CANTAB Task)

  The SST task measures inhibitory control and cognitive flexibility. The SST is a 2-part task. During part 1, an arrow appears on the screen and subjects are asked to press an arrow button on the screen corresponding with the direction of the arrow displayed. During part 2 of the task, subjects follow the same instructions as in part 1; however, they are asked to withhold their response (do not press an arrow button) if they hear an auditory signal (beep) when the arrow is displayed. SST stop signal delay is the length of time between the go stimulus and the stop stimulus at which the subject is able to successfully inhibit their response on 50% of trials. Lower time represents better performance. Observed range for Pooled Active: -31.6 to 48.7 (change from baseline) Observed range for Pooled Placebo: 47.8 to 72.5 (change from baseline)

  Baseline to Part 2 Week 8

Secondary Outcomes (3)

• Change From Baseline in RVP A Prime - Signal Detection Measure of Sensitivity to the Target (CANTAB Task)

  Baseline to Part 2 Week 8

• Change From the Baseline in SWM Strategy 6-8 Boxes - Number of Distinct Boxes Used by the Subject to Begin a New Search for a Token(CANTAB Task)

  Baseline to Part 2 Week 8

• Change From Baseline in SST Proportion of Successful Stops - Measures the Proportion of Completed Stop Trials That Were Successful Stops (CANTAB Task)

  Baseline to Part 2 Week 8

Interventions

Administration of CANTAB and Subject Global Assessment OTHER

At designated visits, subject will perform the CANTAB tasks and complete the Subject Global Assessment Questionnaire

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Subjects with PKU who are currently enrolled in 165-302 Part 2.

You may qualify if:

• 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.

You may not qualify if:

• Any condition that, in the view of the investigator, places the subject at high risk of poor compliance or terminating early from the study

Sponsors & Collaborators

• BioMarin Pharmaceutical: lead

Study Sites (4)

University of Kentucky Medical Center

Lexington, Kentucky, 40536, United States

Location

University of Nebraska Medical Center

Omaha, Nebraska, 68198, United States

Location

University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, 73104, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

Related Links

• BioMarin Pharmaceutical Website
• BioMarin sponsored PKU educational/community website

MeSH Terms

Conditions

Phenylketonurias

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Karen Gu/Director, BioStatistics
• Organization: BioMarin Pharmaceutical Inc.

kgu@bmrn.com

650-829-4017

Study Officials

• Debra Lounsbury

  Principal Scientist/Medical Monitor

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 3, 2015
• First Posted: June 11, 2015
• Study Start: August 5, 2015
• Primary Completion: February 1, 2017
• Study Completion: February 1, 2017
• Last Updated: June 4, 2020
• Results First Posted: June 4, 2020

Record last verified: 2020-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)