Phenylalanine and Its Impact on Cognition

• Status: Recruiting
• Trial end date: 2022-08-01
• Target enrollment: 0
• Participant gender: All

Summary

The PICO-Study is a randomized, placebo-controlled, crossover, non-inferiority trial conducted to add evidence to the current European treatment guidelines for adult patients with phenylketonuria.

• Phase: Phase 4
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: University Hospital Inselspital, Berne
• Collaborator: University of Zurich

Criteria

PATIENTS

Inclusion Criteria:

• PKU diagnosed after a positive newborn screening

• Treatment with Phe-restricted diet starting within the first 30 days of life

• Age ≥18 years

• Capable of following the study design

• Written informed consent

Exclusion Criteria:

• Patients with PKU not following a Phe-restricted diet within 6 months before the study

• Phe concentration above 1600 µmol/L within 6 months before the study

• Concomitant disease states suspected to significantly affect primary or secondary outcomes, e. g. untreated vitamin B12 deficiency

• Known or suspected non-compliance, drug or alcohol abuse

• Change in medications likely to significantly interfere with cognitive function testing

• Known or suspected hypersensitivity or allergy to one of the ingredients of the placebo

• Women who are pregnant or intent to get pregnant during the course of the study or who are breast feeding

• Female participants of childbearing potential, not using and not willing to continue using one (or more) highly efficient (Pearl index less than 1) method of contraception for the entire study duration.

• Inability to follow the procedures of the study, e. g. due to language problems (lack of fluency in German or French), psychological disorders, dementia, etc. of the participant.

• Participation in another interventional study within the 30 days preceding and during the present study.

• Previous enrolment into the current study

• Conditions interfering with MRI such as magnetic (metallic) particles in the skull or brain, cardiac pacemaker, deep brain stimulators, cochlear implant, braces or permanent retainers

HEALTHY CONTROLS

Inclusion Criteria:

• Age ≥18 years

• Comparable to patients with regard to age, gender and educational level

• Capable of following the study design

• Written informed consent

Exclusion Criteria:

• Known or suspected drug or alcohol abuse

• Change in medications likely to significantly interfere with cognitive function testing

• Women who are pregnant or intent to get pregnant during the course of the study or who are breast feeding

• Inability to follow the procedures of the study, e. g. due to language problems (lack of fluency in German or French), psychological disorders, dementia, etc. of the participant.

• Participation in another interventional study within the 30 days preceding and during the present study.

• Previous enrolment into the current study

• Conditions interfering with MRI such as magnetic (metallic) particles in the skull or brain, cardiac pacemaker, deep brain stimulators, cochlear implant, braces or permanent retainers