PKUPKU UPNCT05995717
Evaluation of PKU UP
A study testing PKU UP in people with PKU, run by Vitaflo International, Ltd at 7 sites in United Kingdom.
Trial summary
PKU UP is a prospective, single-arm, open-label, 26-week acceptability study to evaluate PKU UP for the dietary management of participants with phenylketonuria (PKU). Up to 15 participants aged 1 - 10 years old will be recruited and it is anticipated the study will open in at least three sites in the United Kingdom… Read more about this study
- Who is it for?
- Ages 1 year to 10 years
- Conditions the study is for
- PKU
- What is being tested?
- PKU UP
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 7 sitesUnited Kingdom
- Study dates
- 11 Jan 2024to 30 Mar 2026
- 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 1 year to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Diagnosis of PKU requiring a low-protein diet and protein substitute.
- Participants who are adherent with their blood phenylalanine (Phe) testing, in the opinion of the investigator.
- Participants aged 1-10 years.
- Participants who in the opinion of the investigator are anticipated to be able to take at least half of their daily protein equivalent requirement from PKU UP (in order to assess this, a taste test (or similar) may be conducted at investigator's discretion as part of the screening process).
- Willingly given, written, informed consent from the parent(s)/guardian(s).
- Willingly given, written assent by the participant (if appropriate).
Who cannot join
- Inability to comply with the study protocol, in the opinion of the investigator.
- Any co-morbidity, which, in the opinion of the investigator, would preclude participation in the study.
- Diagnosis of persistent hyperphenylalaninemia, or mild PKU not requiring a low protein diet and protein substitute.
- Allergy or intolerance to milk.
- Participants who are currently participating in, plan to participate in or have participated in an interventional investigational drug, food or medical device study within 30 days prior to the screening visit.
- Use of additional micronutrient supplements during the evaluation period, unless clinically indicated and prescribed by the Investigator (must be recorded in both the medical records and case report form).
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United Kingdom7 sites
Royal Belfast Hospital for Sick Children
Belfast, BT12 6BE
Birmingham Children's Hospital
Birmingham, B4 6NH
St. Luke's Hospital
Bradford, BD5 0NA
University Hospitals Bristol and Weston NHS Foundation Trust
Bristol, BS2 8BJ
Great Ormond Street Hospital for Children NHS Foundation Trust
London, WC1N 3JH
Nottingham Children's Hospital
Nottingham, NG7 2UH
Sheffield Children's Hospital
Sheffield, S10 2TH
About this study
Official title: An Acceptability Study to Evaluate the Compliance, Gastrointestinal Tolerance, Palatability and Metabolic Control of Children With Phenylketonuria (PKU) When Using PKU UP (a Food for Special Medical Purposes) as Part of Dietary Management.
PKU UP is a prospective, single-arm, open-label, 26-week acceptability study to evaluate PKU UP for the dietary management of participants with phenylketonuria (PKU). Up to 15 participants aged 1 - 10 years old will be recruited and it is anticipated the study will open in at least three sites in the United Kingdom (UK) to recruit the target number of participants in the required timeframe.
Read the detailed description
PKU is a rare inborn error of metabolism with a prevalence of 1 in 10,000 in the UK population. The mainstay of treatment for most individuals is dietary therapy with a strict protein-restricted diet. We propose to recruit children with a diagnosis of PKU aged 1-10 years.
The study product, PKU UP, is a food for special medical purposes (FSMP), as defined by the Delegated Act EU 2016/128, used for the dietary management of Phenylketonuria (PKU).
The study will be investigating the acceptability of PKU UP, as defined by the Advisory Committee on Borderline Substances (ACBS). This includes the following:
- participant adherence to recommended intakes
- gastrointestinal symptoms
- evaluations of palatability.
Each participant will be on the study for 26 weeks. This includes evaluation part 1 and evaluation part 2:
- Part 1 consists of a 12 week period where participants introduce the study product into their diet. Gastrointestinal tolerance, metabolic control, compliance, product acceptability and dietary quality will be evaluated.
- Part 2 consists of a 14 week period where participants continue to take the study product for a longer assessment of growth and nutritional status.
Data collection will be performed using paper and/or electronic Case Report Forms completed by the investigators at the baseline, evaluation periods and end of study visits. There will also be three questionnaires completed by the parents/guardians over the course of the study, in relation to protein substitute intake, gastrointestinal tolerance, and product acceptability.
- Conditions
- PKU
- Being tested
- PKU UP
- Keywords
- Phenylketonuria, PKU, Glyco-Macro-Peptide, GMP, amino acid, AA, protein substitute, PS, United Kingdom, UK, children, PKU UP, Glycomacropeptide
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PKU UPDietary supplement
PKU UP is a food for special medical purposes (FSMP). This product is for use in the dietary management of Phenylketonuria (PKU) in children aged from one (1) to ten (10) years.
PKU UP is a neutral-flavoured, ready-to-drink, low-phenylalanine protein substitute containing a blend of glycomacropeptide (GMP) isolate, essential and non-essential amino acids, fat, carbohydrate, vitamins, minerals and docosahexaenoic acid (DHA).
| Study group | Role | What they receive |
|---|---|---|
| PKU UP | Experimental | All participants will be assessed by their dietitian and prescribed an appropriate amount of the study product, PKU UP, to manage their phenylketonuria. Receives: Dietary Supplement: PKU UP |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in phe level between baseline and visit 3
Change in phe level as measured by dried blood spot testing
Measured: 26 weeks
Change in tyrosine level between baseline and visit 3
Change in tyrosine level as measured by dried blood spot testing
Measured: 26 weeks
Change in PI impression of metabolic control
Assessment of the participant's metabolic control in the opinion of the investigator
Measured: Week 0, week 6, week 12, week 26
Change in the growth of participants as measured by Height (length in under 2 years of age), Head circumference in under 2 years of age and Weight.
Assessed via anthropometry measurements. Participants will be weighed and measured for growth monitoring.
Measured: Week 0, week 12, week 26
Change in GI tolerance (participant)
Gastrointestinal tolerance assessed using the PedsQL Gastrointestinal Symptoms Scale completed by the participants
Measured: Week 0, week 6, week 12, week 26
Change in PI impression of GI tolerance
Assessment of the participant's GI tolerance in the opinion of the investigator
Measured: Week 0, week 6, week 12, week 26
Change in compliance with study product consumption
Assessed via participant diary regarding study product intake
Measured: Week 0, week 6, week 12, week 26
Change in PI impression of study product intake
Assessment of the participant's compliance in the opinion of the investigator
Measured: Week 0, week 6, week 12, week 26
Change in product acceptability
Assessed via a product acceptability questionnaire completed by the participant/parent
Measured: Week 0, week 12, week 26
Change in dietary intake
Assessed via 24-hour recall and low protein food frequency questionnaire
Measured: Week 0, week 12, week 26
Change in nutritional biochemistry
Assessed via venous blood sample of approximately 10ml
Measured: Week 0, week 26
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Aug 2023
- Study start11 Jan 2024
- Primary completion30 Mar 2026
- Study completion30 Mar 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PKUStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vitaflo International, Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Birmingham Women's and Children's NHS Foundation Trust
- Responsible party
- Sponsor
- Investigators
- Anita MacDonald, Principal investigator, Birmingham Women's and Children's NHS Foundation Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05995717
- Sponsor's protocol ID
- PKU-PAED-LIQ-2022-10-07
- Other IDs
- 327641, 23/NW/0185
- Acronym
- PKU UP
- Status last verified
- May 2026
- First submitted
- 3 Aug 2023
- Last updated
- 18 May 2026
- Results posted
- No
- Data monitoring committee
- No
- 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 18 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Acceptability Study to Evaluate the Compliance, Gastrointestinal Tolerance, Palatability and Metabolic Control of Children With Phenylketonuria (PKU) When Using PKU UP (a Food for Special Medical Purposes) as Part of Dietary Management.
- ClinicalTrials.gov ID
- NCT05995717
- Acronym / sponsor ID
- PKU UP / PKU-PAED-LIQ-2022-10-07
- Conditions
- PKU
- Keywords
- Phenylketonuria, PKU, Glyco-Macro-Peptide, GMP, amino acid, AA, protein substitute, PS
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Vitaflo International, LtdIndustry (pharma, biotech, devices)
- Collaborators
- Birmingham Women's and Children's NHS Foundation Trust
- Study start
- 11 Jan 2024 (actual)
- Primary completion
- 30 Mar 2026 (actual)
- Study completion
- 30 Mar 2026 (actual)
- First posted
- 16 Aug 2023 (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.
PKU UP is a prospective, single-arm, open-label, 26-week acceptability study to evaluate PKU UP for the dietary management of participants with phenylketonuria (PKU). Up to 15 participants aged 1 - 10 years old will be recruited and it is anticipated the study will open in at least three sites in the United Kingdom (UK) to recruit the target number of participants in the required timeframe.
Detailed description
PKU is a rare inborn error of metabolism with a prevalence of 1 in 10,000 in the UK population. The mainstay of treatment for most individuals is dietary therapy with a strict protein-restricted diet. We propose to recruit children with a diagnosis of PKU aged 1-10 years.
The study product, PKU UP, is a food for special medical purposes (FSMP), as defined by the Delegated Act EU 2016/128, used for the dietary management of Phenylketonuria (PKU).
The study will be investigating the acceptability of PKU UP, as defined by the Advisory Committee on Borderline Substances (ACBS). This includes the following:
- participant adherence to recommended intakes
- gastrointestinal symptoms
- evaluations of palatability.
Each participant will be on the study for 26 weeks. This includes evaluation part 1 and evaluation part 2:
- Part 1 consists of a 12 week period where participants introduce the study product into their diet. Gastrointestinal tolerance, metabolic control, compliance, product acceptability and dietary quality will be evaluated.
- Part 2 consists of a 14 week period where participants continue to take the study product for a longer assessment of growth and nutritional status.
Data collection will be performed using paper and/or electronic Case Report Forms completed by the investigators at the baseline, evaluation periods and end of study visits. There will also be three questionnaires completed by the parents/guardians over the course of the study, in relation to protein substitute intake, gastrointestinal tolerance, and product acceptability.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PKU UP | Experimental | Dietary Supplement: PKU UPAll participants will be assessed by their dietitian and prescribed an appropriate amount of the study product, PKU UP, to manage their phenylketonuria. |
Who may qualify
- Ages
- 1 year to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Diagnosis of PKU requiring a low-protein diet and protein substitute.
- Participants who are adherent with their blood phenylalanine (Phe) testing, in the opinion of the investigator.
- Participants aged 1-10 years.
- Participants who in the opinion of the investigator are anticipated to be able to take at least half of their daily protein equivalent requirement from PKU UP (in order to assess this, a taste test (or similar) may be conducted at investigator's discretion as part of the screening process).
- Willingly given, written, informed consent from the parent(s)/guardian(s).
Key exclusion criteria (6 listed)
- Inability to comply with the study protocol, in the opinion of the investigator.
- Any co-morbidity, which, in the opinion of the investigator, would preclude participation in the study.
- Diagnosis of persistent hyperphenylalaninemia, or mild PKU not requiring a low protein diet and protein substitute.
- Allergy or intolerance to milk.
- Participants who are currently participating in, plan to participate in or have participated in an interventional investigational drug, food or medical device study within 30 days prior to the screening visit.
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 |
|---|---|---|
| PKU UPPKU UP is a food for special medical purposes (FSMP). This product is for use in the dietary management of Phenylketonuria (PKU) in children aged from one (1) to ten (10) years. PKU UP is a neutral-flavoured, ready-to-drink, low-phenylalanine protein substitute containing a blend of glycomacropeptide (GMP) isolate, essential and non-essential amino acids, fat, carbohydrate, vitamins, minerals and docosahexaenoic acid (DHA). | Dietary supplement | PKU UP |
Primary outcome
Change in phe level between baseline and visit 3
Time frame: 26 weeks
Change in phe level as measured by dried blood spot testing
Change in tyrosine level between baseline and visit 3
Time frame: 26 weeks
Change in tyrosine level as measured by dried blood spot testing
Change in PI impression of metabolic control
Time frame: Week 0, week 6, week 12, week 26
Assessment of the participant's metabolic control in the opinion of the investigator
Change in the growth of participants as measured by Height (length in under 2 years of age), Head circumference in under 2 years of age and Weight.
Time frame: Week 0, week 12, week 26
Assessed via anthropometry measurements. Participants will be weighed and measured for growth monitoring.
Change in GI tolerance (participant)
Time frame: Week 0, week 6, week 12, week 26
Gastrointestinal tolerance assessed using the PedsQL Gastrointestinal Symptoms Scale completed by the participants
Change in PI impression of GI tolerance
Time frame: Week 0, week 6, week 12, week 26
Assessment of the participant's GI tolerance in the opinion of the investigator
Change in compliance with study product consumption
Time frame: Week 0, week 6, week 12, week 26
Assessed via participant diary regarding study product intake
Change in PI impression of study product intake
Time frame: Week 0, week 6, week 12, week 26
Assessment of the participant's compliance in the opinion of the investigator
Change in product acceptability
Time frame: Week 0, week 12, week 26
Assessed via a product acceptability questionnaire completed by the participant/parent
Change in dietary intake
Time frame: Week 0, week 12, week 26
Assessed via 24-hour recall and low protein food frequency questionnaire
Change in nutritional biochemistry
Time frame: Week 0, week 26
Assessed via venous blood sample of approximately 10ml
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 18 May 2026.
- Individual participant dataSharing planned: No
Related trials for PKU
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RecruitingPhase 2
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Impact of Phenylalanine Elevations on Brain and Cognition in Adult PKU Carriers
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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.
Anita MacDonald principal investigator
Birmingham Women's and Children's NHS Foundation Trust
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing PKU UP in people with PKU, run by Vitaflo International, Ltd at 7 sites in United Kingdom.
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 6 inclusion and 6 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 18 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in 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?
Change in phe level between baseline and visit 3 (time frame: 26 weeks). The study lists 11 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 18 May 2026.