PhenylketonuriasNCT04210206
Short-term Effects of Good Metabolic Control on Cognitive Function, Wellbeing, and Metabolic Parameters in Adult Patients With Phenylketonuria
A study testing PHE-diet in people with Phenylketonurias, run by Klaus Parhofer at 1 site in Germany.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 59 years
- Conditions the study is for
- Phenylketonurias
- What is being tested?
- PHE-diet
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 1 siteGermany
- Study dates
- 1 Dec 2019to 31 Aug 2023
- Phase
- Interventional
- 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 18 to 59 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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
Who cannot join
- Reduced cognitive function (unability to perform test battery)
- Drug and or alcohol abuse
- Treatment with BH4
- Treatment with drugs known to affect cognitive function
Trial locations and contacts
1 site in 1 country.
About this study
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)
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.
- Conditions
- Phenylketonurias
- Being tested
- PHE-diet
- Keywords
- cognitive function adults
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PHE-dietOther
phenylalanine-restricted diet with phenylalanine free supplements for 8 weeks
| Study group | Role | What they receive |
|---|---|---|
| Diet | Experimental | Not described Receives: Other: PHE-diet |
| Control | No intervention | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Spatial working memory strategy index
cognitive function; scores range from 4 to 28, with lower scores representing better outcome
Measured: 8 weeks
Secondary outcomes (3)
Reaction time
cognitive function; milliseconds, with faster times representing better outcome
Measured: 8 weeks
Paired Associates Learning
cognitive function; scores range from 0 to 70, with lower scores indicating better outcome
Measured: 8 weeks
Multitasking Test
cognitive function; scores range from 0 to 279, with lower scores indicating better outcome
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Dec 2019
- Study start1 Dec 2019
- Primary completion31 Aug 2023
- Study completion31 Aug 2023
Why it stopped: Recruitment failure
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PhenylketonuriasStudies 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Klaus Parhofer
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Klaus Parhofer, Ludwig-Maximilians - University of Munich
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04210206
- Sponsor's protocol ID
- CoFu-PKU
- Status last verified
- Dec 2023
- First submitted
- 19 Dec 2019
- Last updated
- 14 Dec 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT04210206
- Acronym / sponsor ID
- CoFu-PKU
- Conditions
- Phenylketonurias
- Keywords
- cognitive function adults
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Recruitment failure
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Klaus ParhoferUniversity, hospital or other
- Study start
- 1 Dec 2019 (actual)
- Primary completion
- 31 Aug 2023 (actual)
- Study completion
- 31 Aug 2023 (actual)
- First posted
- 24 Dec 2019 (actual)
- 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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Diet | Experimental | Other: PHE-diet |
| Control | No intervention | – |
Who may qualify
- Ages
- 18 to 59 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (4 listed)
- Reduced cognitive function (unability to perform test battery)
- Drug and or alcohol abuse
- Treatment with BH4
- Treatment with drugs known to affect cognitive function
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PHE-dietphenylalanine-restricted diet with phenylalanine free supplements for 8 weeks | Other | Diet |
Primary outcome
Spatial working memory strategy index
Time frame: 8 weeks
cognitive function; scores range from 4 to 28, with lower scores representing better outcome
3 secondary outcomes
Reaction time
Time frame: 8 weeks
Paired Associates Learning
Time frame: 8 weeks
Multitasking Test
Time frame: 8 weeks
Results and safety
The study was stopped early (reason given: Recruitment failure). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Dec 2023.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing PHE-diet in people with Phenylketonurias, run by Klaus Parhofer 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 5 inclusion and 4 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: stopped early, as of the sponsor's last update on 14 Dec 2023. 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 was stopped early (reason given: Recruitment failure). Results are not available in the source record.
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.
What does the primary outcome measure?
Spatial working memory strategy index (time frame: 8 weeks). It is what the study was designed to measure, not a finding.
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 14 Dec 2023.