Clinical trial NCT02468570: 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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Phenylketonuria
Keywords: PKU
PEG-PAL
Pegvaliase
BMN 165
rAv-PAL PEG
PRISM 302
PRISM 303
BioMarin
165-302
Lead sponsor: BioMarin Pharmaceutical
Sponsor class: INDUSTRY
First posted: 2015-06-11
Enrollment: 9
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Subjects with PKU who are currently enrolled in 165-302 Part 2.

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.

Detailed description: Study 165-303, is a Phase 3 substudy enrolling approximately 100 subjects, aged ≥ 18 to ≤ 70 years old, with PKU who are participating in the Phase 3 study, 165-302.

Per the 165-303 protocol, the investigator does not assign a specific intervention to the study participants. In accordance with the protocol for Study 165-303, subjects were asked to perform selected set of three tasks from the CANTAB tool (Rapid Visual Processing \[RVP\], Spatial Working Memory \[SWM\], and Stop Signal Task \[SST\]) to assess executive function and each subject's self perception of their current state was measured using a subject global assessment questionnaire that contains seven questions about current state perception of attention, energy level, tiredness, confusion, sadness, anger and tension.

The time points for administering the above tools are as follows, which coincide with time points in part 2 and part 4 of Study 165-302: screening (entry into Part 2 165-302 Day 1 of Week 1), baseline visit (Part 2 Week 8), and 3 additional study visits at different time points in the Part 4 of 165-302 study.

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

Primary outcomes:
measure: Change From Baseline in Rapid Visual Processing (RVP) Mean Response Latency (CANTAB Task)
timeFrame: Baseline to Part 2 Week 8
measure: Change From Baseline in Spatial Working Memory (SWM) Between Errors 4-8 Boxes (CANTAB Task)
timeFrame: Baseline to Part 2 Week 8
measure: Change From Baseline in Stop Signal Task (SST) Stop Signal Reaction T (CANTAB Task)
timeFrame: Baseline to Part 2 Week 8
Secondary outcomes:
measure: Change From Baseline in RVP A Prime - Signal Detection Measure of Sensitivity to the Target (CANTAB Task)
timeFrame: Baseline to Part 2 Week 8
measure: 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)
timeFrame: Baseline to Part 2 Week 8
measure: Change From Baseline in SST Proportion of Successful Stops - Measures the Proportion of Completed Stop Trials That Were Successful Stops (CANTAB Task)
timeFrame: Baseline to Part 2 Week 8
Other outcomes:
Locations:
city: Lexington
country: United States
facility: University of Kentucky Medical Center
state: Kentucky
city: Omaha
country: United States
facility: University of Nebraska Medical Center
state: Nebraska
city: Oklahoma City
country: United States
facility: University of Oklahoma Health Sciences Center
state: Oklahoma
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
Study officials:
affiliation: Principal Scientist/Medical Monitor
name: Debra Lounsbury
role: STUDY_DIRECTOR
References: