A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302 (PRISM303)

BioMarin Pharmaceutical

Status: Completed

Conditions: Phenylketonuria

Treatments: Other: Administration of CANTAB and Subject Global Assessment

Study type: Observational

Funder types: Industry

Identifiers

NCT02468570

165-303

Details and patient eligibility

About

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.

Full 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.

Enrollment: 9 patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

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

Trial design