Follow-up of Adult Phenylketonuria (PKU) Patients

September 9, 2013 updated by: Koletzko - Office, Ludwig-Maximilians - University of Munich

5-year Follow-up of the Comparison of Life and Physical Health in Adult Patients With PKU and Healthy Age Matched Controls

Adult patients with phenylketonuria (PKU) at the age around 40 years belong to the first patients generation with early treatment of the disease. PKU is caused by an inborn error of the amino acid metabolism and the so far best suitable therapy is an early and strict diet, which is low in phenylalanine.

Besides an early and continuously treatment in childhood, the nutritional and medical support during adolescence and adulthood have been suggested to influence the long-term physical health of adult PKU patients. As many adult PKU patients tend to neglect the necessarily strict diet, they do not get a balanced diet. For PKU patients some nutrients, which may be rare in an unbalanced diet, might help to improve health status, physical and neurological performance and quality of life.

Information about the longitudinal development of the patients status and the influence of the type of their medical care is not available. In this 5 year follow-up the investigators aim to study the quality of life and the medical, nutritional and psychological status of adult PKU patients, in whom corresponding information has already been collected previously.

Study Overview

Status

Completed

Conditions

Study Type

Observational

Enrollment (Actual)

40

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Germany
      • Muenchen, Germany, D-80337
        • Dr. von Hauner Childrens Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

25 years to 70 years (Adult, Older Adult)

Accepts Healthy Volunteers

Yes

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Adult PKU patients who are or have been attending the Departements of Metabolic Diseases of the Klinikum der Universität München will be recruited for this study, if they had been participating already in the last study trial comparing adult PKU patients with healthy controls.

It will be attempted to include the same control persons as in the last study trial.

Description

Inclusion Criteria:

  • assured clinical diagnose of PKU or proof of mutation in the phenylalanine hydroxylase gene (for PKU patients only)
  • both groups need to hand in a signed consent to participate in the study
  • controls: have to be omnivores and should not be on drugs, which influence the lipid metabolism, during the last 3 months
  • attendance of the last study trial

Exclusion Criteria:

  • not fulfill the inclusion criteria

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
healthy controls
PKU patients

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ludwig-Maximilians - University of Munich

Investigators

  • Principal Investigator: Berthold Koletzko, Prof., Ludwig-Maximilians - University of Munich

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

March 1, 2010

Primary Completion (Actual)

September 1, 2012

Study Completion (Actual)

September 1, 2013

Study Registration Dates

First Submitted

March 30, 2010

First Submitted That Met QC Criteria

March 30, 2010

First Posted (Estimate)

March 31, 2010

Study Record Updates

Last Update Posted (Estimate)

September 10, 2013

Last Update Submitted That Met QC Criteria

September 9, 2013

Last Verified

September 1, 2013

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • A-PKU2010

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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