Short-term Effects of Good Metabolic Control on Cognitive Function, Wellbeing, and Metabolic Parameters in Adult Patients With Phenylketonuria

Klaus Parhofer

Status: Terminated

Conditions: Phenylketonurias

Treatments: Other: PHE-diet

Study type: Interventional

Funder types: Other

Identifiers

NCT04210206

CoFu-PKU

Details and patient eligibility

About

The goal of this study is to evaluate if in adult patients with phenylketonuria (PKU) without strict metabolic control during the last 12 months, strict metabolic control for 8 weeks results in an improvement of cognitive function.

Enrollment: 13 patients

Sex: All

Ages: 18 to 59 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Men and women with classical PKU
• Age ≥ 18 years
• Good metabolic control during childhood (at least until 12 years of age)
• Plasma Phe levels ≥ 15 mg/dl for ≥ 1 year
• Written informed consent prior to study participation

Exclusion criteria

• Reduced cognitive function (unability to perform test battery)
• Drug and or alcohol abuse
• Treatment with BH4
• Treatment with drugs known to affect cognitive function

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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13 participants in 2 patient groups

Diet

Experimental group

Treatment:

Other: PHE-diet

Control

No Intervention group