Nutritional DeficienciesNCT01096758
Follow-up of Adult Phenylketonuria (PKU) Patients
An observational study of Nutritional Deficiencies and Phenylketonuria, run by Ludwig-Maximilians - University of Munich at 1 site in Germany.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 25 to 70 yearsHealthy volunteers can take part
- Conditions the study is for
- Nutritional Deficiencies, Phenylketonuria
- How many people
- 40 participants
- Where
- 1 siteGermany
- Study dates
- Mar 2010to Sep 2013
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 25 to 70 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
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.
Who may be able to join
- 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
Who cannot join
- not fulfill the inclusion criteria
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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.
- Conditions
- Nutritional Deficiencies, Phenylketonuria
- Keywords
- PKU, phenylketonuria, adult, patient, development, nutritional deficiencies, neurological outcome, psychological and neurological development, comparison with healthy adults, longitudinal development, quality of life
- Medical subject headings
- Malnutrition, Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| PKU patients | Not described | |
| healthy controls | Not described |
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Mar 2010 (estimated)
- Study startMar 2010
- Primary completionSep 2012
- Study completionSep 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Nutritional DeficienciesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ludwig-Maximilians - University of Munich
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Koletzko - Office, Ludwig-Maximilians - University of Munich
- Investigators
- Berthold Koletzko, Prof., Principal investigator, Ludwig-Maximilians - University of Munich
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01096758
- Sponsor's protocol ID
- A-PKU2010
- Status last verified
- Sep 2013
- First submitted
- 30 Mar 2010
- Last updated
- 10 Sep 2013
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Sep 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 5-year Follow-up of the Comparison of Life and Physical Health in Adult Patients With PKU and Healthy Age Matched Controls
- ClinicalTrials.gov ID
- NCT01096758
- Acronym / sponsor ID
- A-PKU2010
- Conditions
- Nutritional Deficiencies, Phenylketonuria
- Keywords
- PKU, phenylketonuria, adult, patient, development, nutritional deficiencies, neurological outcome, psychological and neurological development
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Ludwig-Maximilians - University of MunichUniversity, hospital or other
- Study start
- Mar 2010
- Primary completion
- Sep 2012 (actual)
- Study completion
- Sep 2013 (actual)
- First posted
- 31 Mar 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| PKU patients | – | – |
| healthy controls | – | – |
Who may qualify
- Ages
- 25 to 70 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (1 listed)
- not fulfill the inclusion criteria
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Sep 2013.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Berthold Koletzko, Prof. principal investigator
Ludwig-Maximilians - University of Munich
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Nutritional Deficiencies and Phenylketonuria, run by Ludwig-Maximilians - University of Munich at 1 site in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Sep 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Sep 2013.