N/A
N=9
A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302
Phenylketonuria
Bottom Line
View on ClinicalTrials.gov: NCT02468570 ↗Enrolled (actual)
9
Serious AEs
—
Results posted
Jun 2020
Primary outcome: Primary: Change From Baseline in Rapid Visual Processing (RVP) Mean Response Latency (CANTAB Task) — -24.23; 18.03 Millisec
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- Administration of CANTAB and Subject Global Assessment (Other)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- BioMarin Pharmaceutical
- Primary completion
- Feb 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Rapid Visual Processing (RVP) Mean Response Latency (CANTAB Task) |
-24.23; 18.03 | — |
| PRIMARY Change From Baseline in Spatial Working Memory (SWM) Between Errors 4-8 Boxes (CANTAB Task) |
-4.2; 3.0 | — |
| PRIMARY Change From Baseline in Stop Signal Task (SST) Stop Signal Reaction T (CANTAB Task) |
8.42; 58.93 | — |
| SECONDARY Change From Baseline in RVP A Prime - Signal Detection Measure of Sensitivity to the Target (CANTAB Task) |
0.0117; -0.0247 | — |
| SECONDARY 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) |
-0.2; -1.0 | — |
| SECONDARY Change From Baseline in SST Proportion of Successful Stops - Measures the Proportion of Completed Stop Trials That Were Successful Stops (CANTAB Task) |
-0.0333; 0.0 | — |
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.
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
Data sourced from ClinicalTrials.gov (NCT02468570). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.