Status:
COMPLETED
Phenylalanine and Its Impact on Cognition
Lead Sponsor:
Insel Gruppe AG, University Hospital Bern
Collaborating Sponsors:
University of Zurich
Conditions:
Phenylketonuria
Eligibility:
All Genders
18+ years
Phase:
PHASE4
Brief 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.
Detailed Description
Phenylketonuria (PKU) is a rare autosomal recessive disorder caused by deficiency of the phenylalanine hydroxylase enzyme leading to an impaired conversion of the amino acid phenylalanine (Phe) to tyr...
Eligibility Criteria
Inclusion
- PATIENTS
- 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
- 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
Key Trial Info
Start Date :
August 19 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 17 2022
Estimated Enrollment :
90 Patients enrolled
Trial Details
Trial ID
NCT03788343
Start Date
August 19 2019
End Date
June 17 2022
Last Update
March 7 2023
Active Locations (1)
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1
Department of Diabetes, Endocrinology, Nutritional Medicine and Metabolism (UDEM), Inselspital, Bern University Hospital
Bern, Switzerland, 3010