Phenylketonuria (PKU)PANDANCT04086511
PANDA: PKU Amino Acid Evaluation
An observational study of Phenylketonuria (PKU), run by Nutricia Research at 2 sites in 2 countries.
Trial summary
Phenylketonuria (PKU) is a rare inherited metabolic disorder, where subjects are born with a genetic deficiency in the phenylalanine hydroxylase enzyme (PAH), which leaves them unable to convert Phenylalanine (Phe) into Tyrosine (Tyr). Read more about this study
- Who is it for?
- Ages 2 to 12 years
- Conditions the study is for
- Phenylketonuria (PKU)
- How many people
- 37 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 10 Sep 2019to 14 Apr 2023
- 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 2 to 12 years
- All sexes
- Child
Study population: Children with PKU and age- and sex-matched non-PKU comparison subjects.
Who may be able to join
- Both PKU and Non-PKU comparison subjects:
- Age ≥ 2 and ≤ 12 years
- Willing and able to provide informed consent by parents or legal representatives (and assent if required by local law/regulations)
- One subject per family
- PKU subjects specific inclusion criteria:
- PKU subjects identified by newborn screening and started low Phe diet before 1 month age
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- Usage of at least two Phe-free protein substitutes on a daily basis for at least 26 consecutive weeks up to Visit 1
- Average Phe-level ≤360 µmol/L based on at least two blood Phe values from the past 12 months up to Visit 1
- Non-PKU comparison subjects specific inclusion criteria:
- Same age (±3 years) and sex as an included PKU subject
Who cannot join
- Both PKU and Non-PKU comparison subjects:
- Current psychiatric disorders
- Severe hepatic, thyroid or renal dysfunction
- Acute illnesses like fever, flu, diarrhea, or vomiting (subjects should be symptom free for a week prior to V1)
- Serious conditions (e.g. cancer, hydrocephalus, fatal heart disease)
- Participation in any other clinical intervention studies involving test products concomitantly or within six weeks prior to entry into the study
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- PKU specific exclusion criteria:
- Use of BH4, or drugs that may interfere with main outcomes
- Non-PKU comparison subjects specific exclusion criteria:
- PKU diagnosis or any other diagnosed disorder of amino or organic acid metabolism
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
Official title: PANDA: A Cross-sectional Study to Measure Blood Amino Acid Levels in PKU Children on a Protein Substitute
Phenylketonuria (PKU) is a rare inherited metabolic disorder, where subjects are born with a genetic deficiency in the phenylalanine hydroxylase enzyme (PAH), which leaves them unable to convert Phenylalanine (Phe) into Tyrosine (Tyr). PKU patients have specific dietary needs and must follow a restrictive diet in the aim of preventing toxic levels of the amino acid phenylalanine (Phe) accumulation.
Read the detailed description
The exploratory study's main objective is to measure blood amino acid levels and to gain quantitative insights in children with PKU on a protein substitute with respect to evaluation of nutritional intake.
- Conditions
- Phenylketonuria (PKU)
- Medical subject headings
- 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 |
|---|---|---|
| Children with PKU | Not described | |
| Age- and sex-matched non-PKU comparison subjects | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Amino acid levels in blood
Measuring amino acid levels in blood [μmol/L]
Measured: day 1
Nutrient Intake
Measured by three-day diet diary. Nutrients in [mg/day]
Measured: day 1 - day 3
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Sep 2019
- Study start10 Sep 2019
- Primary completion21 Dec 2022
- Study completion14 Apr 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Phenylketonuria (PKU)Studies 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
- Other
- Time perspective
- Cross-sectional
- Enrolment
- 37 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nutricia Research
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Dr. P. Verloo, Principal investigator, University Hospital, Ghent
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04086511
- Sponsor's protocol ID
- MBB18TA18444
- Acronym
- PANDA
- Status last verified
- Sep 2021
- First submitted
- 10 Sep 2019
- Last updated
- 2 Jun 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PANDA: A Cross-sectional Study to Measure Blood Amino Acid Levels in PKU Children on a Protein Substitute
- ClinicalTrials.gov ID
- NCT04086511
- Acronym / sponsor ID
- PANDA / MBB18TA18444
- Conditions
- Phenylketonuria (PKU)
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 37 participants (actual)
- Lead sponsor
- Nutricia ResearchIndustry (pharma, biotech, devices)
- Study start
- 10 Sep 2019 (actual)
- Primary completion
- 21 Dec 2022 (actual)
- Study completion
- 14 Apr 2023 (actual)
- First posted
- 11 Sep 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.
Phenylketonuria (PKU) is a rare inherited metabolic disorder, where subjects are born with a genetic deficiency in the phenylalanine hydroxylase enzyme (PAH), which leaves them unable to convert Phenylalanine (Phe) into Tyrosine (Tyr). PKU patients have specific dietary needs and must follow a restrictive diet in the aim of preventing toxic levels of the amino acid phenylalanine (Phe) accumulation.
Detailed description
The exploratory study's main objective is to measure blood amino acid levels and to gain quantitative insights in children with PKU on a protein substitute with respect to evaluation of nutritional intake.
Study design
- Observational model
- Other
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Children with PKU | – | – |
| Age- and sex-matched non-PKU comparison subjects | – | – |
Who may qualify
- Ages
- 2 to 12 years
- Sex
- All
- Age groups
- Child
- Study population
- Children with PKU and age- and sex-matched non-PKU comparison subjects.
Key inclusion criteria (10 listed)
- Age ≥ 2 and ≤ 12 years
- Willing and able to provide informed consent by parents or legal representatives (and assent if required by local law/regulations)
- One subject per family
- PKU subjects identified by newborn screening and started low Phe diet before 1 month age
- Usage of at least two Phe-free protein substitutes on a daily basis for at least 26 consecutive weeks up to Visit 1
Key exclusion criteria (10 listed)
- Current psychiatric disorders
- Severe hepatic, thyroid or renal dysfunction
- Acute illnesses like fever, flu, diarrhea, or vomiting (subjects should be symptom free for a week prior to V1)
- Serious conditions (e.g. cancer, hydrocephalus, fatal heart disease)
- Participation in any other clinical intervention studies involving test products concomitantly or within six weeks prior to entry into the study
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
Amino acid levels in blood
Time frame: day 1
Measuring amino acid levels in blood [μmol/L]
Nutrient Intake
Time frame: day 1 - day 3
Measured by three-day diet diary. Nutrients in [mg/day]
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 2 Jun 2023.
- Individual participant dataSharing planned: No
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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.
Dr. P. Verloo principal investigator
University Hospital, Ghent
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Phenylketonuria (PKU), run by Nutricia Research at 2 sites in 2 countries.
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 10 inclusion and 10 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 2 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Belgium, United Kingdom. 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.
What does the primary outcome measure?
Amino acid levels in blood (time frame: day 1). The study lists 2 primary outcomes in all. 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 2 Jun 2023.