NCT04210206

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.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
13

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Dec 2019

Longer than P75 for not_applicable

Geographic Reach
1 country

1 active site

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 1, 2019

Completed
18 days until next milestone

First Submitted

Initial submission to the registry

December 19, 2019

Completed
5 days until next milestone

First Posted

Study publicly available on registry

December 24, 2019

Completed
3.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 31, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 31, 2023

Completed
Last Updated

December 14, 2023

Status Verified

December 1, 2023

Enrollment Period

3.8 years

First QC Date

December 19, 2019

Last Update Submit

December 8, 2023

Conditions

Phenylketonurias

Keywords

cognitive function adults

Outcome Measures

Primary Outcomes (1)

  • Spatial working memory strategy index

    cognitive function; scores range from 4 to 28, with lower scores representing better outcome

    8 weeks

Secondary Outcomes (3)

  • Reaction time

    8 weeks

  • Paired Associates Learning

    8 weeks

  • Multitasking Test

    8 weeks

Study Arms (2)

Diet

EXPERIMENTAL
Other: PHE-diet

Control

NO INTERVENTION

Interventions

PHE-dietOTHER

phenylalanine-restricted diet with phenylalanine free supplements for 8 weeks

Diet

Eligibility Criteria

Age18 Years - 59 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64)

You may qualify if:

  • 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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University Munich

Munich, 81377, Germany

Location

MeSH Terms

Conditions

Phenylketonurias

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesAmino Acid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Study Design

Study Type
interventional
Phase
not applicable
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Professor of Endocrinology and Metabolism

Study Record Dates

First Submitted

December 19, 2019

First Posted

December 24, 2019

Study Start

December 1, 2019

Primary Completion

August 31, 2023

Study Completion

August 31, 2023

Last Updated

December 14, 2023

Record last verified: 2023-12

Locations

DE(1)