Short-term Effects of Good Metabolic Control on Cognitive... | NCT04210206

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

NCT04210206

Sponsor

Klaus Parhofer

Status

Terminated

Last Update Posted

Dec 14, 2023Actual

Enrollment

13Actual

Phase

Not Applicable

Conditions

Phenylketonurias

Interventions

PHE-diet

Countries

Germany

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

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)

Status Module

Record Verification Date

Dec 2023

Overall Recruitment Status or Expanded Access Status

Terminated

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Recruitment failure

Expanded Access Info

Start Date

Dec 1, 2019Actual

Primary Completion Date

Aug 31, 2023Actual

Completion Date

Aug 31, 2023Actual

First Submitted Date

Dec 19, 2019

First Submission Date that Met QC Criteria

Dec 20, 2019

First Posted Date

Dec 24, 2019Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Dec 8, 2023

Last Update Posted Date

Dec 14, 2023Actual

Sponsor/Collaborators Module

Responsible Party, by Official Title

Klaus Parhofer, Professor of Endocrinology and Metabolism, Ludwig-Maximilians - University of MunichSponsor-Investigator

Lead Sponsor

Klaus ParhoferOTHER

Collaborators

Not provided

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not Applicable

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

13Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Diet	Experimental		Other: PHE-diet
Control	No Intervention

Interventions

Name	Type	Description	Arm Group Labels	Other Names
PHE-diet	Other	phenylalanine-restricted diet with phenylalanine free supplements for 8 weeks	Diet

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Spatial working memory strategy index	cognitive function; scores range from 4 to 28, with lower scores representing better outcome	8 weeks

Secondary Outcomes

Measure	Description	Time Frame
Reaction time	cognitive function; milliseconds, with faster times representing better outcome	8 weeks
Paired Associates Learning	cognitive function; scores range from 0 to 70, with lower scores indicating better outcome

Other Outcomes

Not provided

Eligibility Module

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

59 Years

Standard Ages

Adult

Study Population

Not provided

Sampling Method

Not provided

Facility	Status	City	State	ZIP	Country	Contacts
University Munich		Munich		81377	Germany

Condition Browse Module

MeSH Terms

ID	Term
D010661	Phenylketonurias

Ancestor Terms

ID	Term
D020739	Brain Diseases, Metabolic, Inborn
D001928	Brain Diseases, Metabolic
D001927	Brain Diseases
D002493	Central Nervous System Diseases

Browse Leaves

Not provided

Browse Branches

Not provided