Back to resultsShort-term Effects of Good Metabolic Control on Cognitive Function, Wellbeing, and Metabolic Parameters in Adult Patients With Phenylketonuria
Primary Purpose
Phenylketonurias
Status
Recruiting
Phase
Not Applicable
Locations
Germany
Study Type
Interventional
Intervention
PHE-diet
Sponsored by
About this trial
This is an interventional treatment trial for Phenylketonurias focused on measuring cognitive function adults
Eligibility Criteria
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
Sites / Locations
- University MunichRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
No Intervention
Arm Label
Diet
Control
Arm Description
Outcomes
Primary Outcome Measures
Spatial working memory strategy index
cognitive function; scores range from 4 to 28, with lower scores representing better outcome
Secondary Outcome Measures
Reaction time
cognitive function; milliseconds, with faster times representing better outcome
Paired Associates Learning
cognitive function; scores range from 0 to 70, with lower scores indicating better outcome
Multitasking Test
cognitive function; scores range from 0 to 279, with lower scores indicating better outcome
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04210206
Brief Title
Short-term Effects of Good Metabolic Control on Cognitive Function, Wellbeing, and Metabolic Parameters in Adult Patients With Phenylketonuria
Official Title
Kurzfristiger Einfluss Einer Guten Stoffwechseleinstellung Auf Kognitive Funktion, Wohlbefinden Und Stoffwechselparameter Bei Erwachsenen Patienten Mit Phenylketonurie (Short-term Effects of Good Metabolic Control on Cognitive Function, Wellbeing, and Metabolic Parameters in Adult Patients With Phenylketonuria)
Study Type
Interventional
2. Study Status
Record Verification Date
April 2022
Overall Recruitment Status
Recruiting
Study Start Date
December 1, 2019 (Actual)
Primary Completion Date
December 2022 (Anticipated)
Study Completion Date
December 2022 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Klaus Parhofer
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Phenylketonurias
Keywords
cognitive function adults
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
36 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Diet
Arm Type
Experimental
Arm Title
Control
Arm Type
No Intervention
Intervention Type
Other
Intervention Name(s)
PHE-diet
Intervention Description
phenylalanine-restricted diet with phenylalanine free supplements for 8 weeks
Primary Outcome Measure Information:
Title
Spatial working memory strategy index
Description
cognitive function; scores range from 4 to 28, with lower scores representing better outcome
Time Frame
8 weeks
Secondary Outcome Measure Information:
Title
Reaction time
Description
cognitive function; milliseconds, with faster times representing better outcome
Time Frame
8 weeks
Title
Paired Associates Learning
Description
cognitive function; scores range from 0 to 70, with lower scores indicating better outcome
Time Frame
8 weeks
Title
Multitasking Test
Description
cognitive function; scores range from 0 to 279, with lower scores indicating better outcome
Time Frame
8 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
59 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Klaus G Parhofer, MD
Phone
+4989440073010
Email
klaus.parhofer@med.uni-muenchen.de
Facility Information:
Facility Name
University Munich
City
Munich
ZIP/Postal Code
81377
Country
Germany
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Klaus G Parhofer, MD
Phone
+49-89-7095
Ext
3010
Email
klaus.parhofer@med.uni-muenchen.de
First Name & Middle Initial & Last Name & Degree
Elisa Waldmann, MD
Phone
+49-89-7095
Ext
2262
Email
elisa.waldmann@med.uni-muenchen.de
12. IPD Sharing Statement
Learn more about this trial
Short-term Effects of Good Metabolic Control on Cognitive Function, Wellbeing, and Metabolic Parameters in Adult Patients With Phenylketonuria
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