Phenylketonuria (PKU)PEMP-ATNCT06694662
PKU EASY Microtabs Plus - Acceptability and Tolerance
A study testing PKU EASY Microtabs Plus in people with Phenylketonuria (PKU), run by Galen Limited at 1 site in United Kingdom.
Trial summary
PEMP-AT is a prospective, single-arm, open-label, 1-week acceptability study to evaluate PKU EASY Microtabs Plus for the dietary management of participants with phenylketonuria (PKU). Up to 10 participants aged 3 years and above will be recruited in a single-centre in the UK. Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Phenylketonuria (PKU)
- What is being tested?
- PKU EASY Microtabs Plus
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 15 Mar 2025to 13 Aug 2025
- 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 3 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of PKU requiring an appropriate protein substitute
- Subjects who are already taking an appropriate protein substitute and are willing to try the study product for 7 days
- Subjects aged 3 years and above
- Written informed consent obtained from subject or parent/legal guardian, depending on subject age
Who cannot join
- Presence of serious concurrent illness
- Lead Dietitian's uncertainty about the willingness or ability of the patient to comply with the protocol requirements
- Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study, with the exception of sapropterin
- Any subject having taken antibiotics over the previous 2 weeks leading up to the study
- Subjects less than 3 years of age
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
Birmingham Children's Hospital
Birmingham, UK, B4 6NH
About this study
PEMP-AT is a prospective, single-arm, open-label, 1-week acceptability study to evaluate PKU EASY Microtabs Plus for the dietary management of participants with phenylketonuria (PKU). Up to 10 participants aged 3 years and above will be recruited in a single-centre in the UK.
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 3 years and above.
The study product, PKU EASY Microtabs Plus 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 EASY Microtabs Plus, 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 1 week.
The study consists of a 1 week period where participants introduce the study product into their diet. Gastrointestinal tolerance, compliance and product acceptability will be evaluated.
Data collection will be performed using paper data collection forms completed by the investigators at the baseline and end of study visits. Patients/parents/guardians, as appropriate, will also complete a brief daily questionnaire on each of the 7 study days. There will also be a questionnaire completed by the patient/parents/guardians over the course of the study, in relation to protein substitute intake, gastrointestinal tolerance, and product acceptability.
- Conditions
- Phenylketonuria (PKU)
- Being tested
- PKU EASY Microtabs Plus
- Keywords
- PKU, Microtabs, protein substitute, amino acid
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PKU EASY Microtabs PlusDietary supplement
PKU EASY Microtabs Plus is a food for special medical purposes (FSMP). This product is for use in the dietary management of Phenylketonuria (PKU) in patients from 3 years of age upwards.
PKU EASY Microtabs Plus is a microtablet form, slow-release, phenylalanine-free protein substitute containing a blend of essential and non-essential amino acids, vitamins, minerals and trace elements.
| Study group | Role | What they receive |
|---|---|---|
| Participant Group | Experimental | All participants will be assessed by their dietitian and prescribed an appropriate amount of the study product, PKU EASY Microtabs Plus, to manage their phenylketonuria. Receives: Dietary Supplement: PKU EASY Microtabs Plus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in GI tolerance (participant)
Gastrointestinal tolerance assessed using symptom and severity questionnaire completed by the participants. Severity of symptoms will be rated on a scale of 0 - 3, with 0 being no symptoms and 3 being severe or very troublesome symptoms.
Measured: Week 0, week 1
Rate of patient compliance with consumption of the prescribed volume of study product
Assessed via participant diary regarding study product intake. The quantity of study product actually consumed vs the prescribed amount will be recorded. The reason for not consuming the full dose will be recorded by the patient/parent/guardian as free text.
Measured: Week 1
Change in product acceptability
Assessed via a product acceptability questionnaire completed by the participant/parent/guardian. Organoleptic properties, such as taste and texture, of the study product will be assessed on a rating scale of terrible to great.
Measured: Week 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2024
- Study start15 Mar 2025
- Primary completion13 Aug 2025
- Study completion13 Aug 2025
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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Galen Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Birmingham Women's and Children's NHS Foundation Trust
- Responsible party
- Sponsor
- Investigators
- Anita MacDonald, Ph.D., Principal investigator, Birmingham Women's and Children's NHS Foundation Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06694662
- Sponsor's protocol ID
- PKU-004
- Acronym
- PEMP-AT
- Status last verified
- Nov 2025
- First submitted
- 12 Nov 2024
- Last updated
- 26 Nov 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Due to the very small number of participants and rarity of the condition being tested the ability to maintain participant pseudonymity could be compromised.
Study at a glance
From the registry record, last updated by the sponsor on 26 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PKU EASY Microtabs Plus - Acceptability and Tolerance
- ClinicalTrials.gov ID
- NCT06694662
- Acronym / sponsor ID
- PEMP-AT / PKU-004
- Conditions
- Phenylketonuria (PKU)
- Keywords
- PKU, Microtabs, protein substitute, amino acid
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Galen LimitedIndustry (pharma, biotech, devices)
- Collaborators
- Birmingham Women's and Children's NHS Foundation Trust
- Study start
- 15 Mar 2025 (actual)
- Primary completion
- 13 Aug 2025 (actual)
- Study completion
- 13 Aug 2025 (actual)
- First posted
- 19 Nov 2024 (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.
PEMP-AT is a prospective, single-arm, open-label, 1-week acceptability study to evaluate PKU EASY Microtabs Plus for the dietary management of participants with phenylketonuria (PKU). Up to 10 participants aged 3 years and above will be recruited in a single-centre in the UK.
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 3 years and above.
The study product, PKU EASY Microtabs Plus 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 EASY Microtabs Plus, 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 1 week.
The study consists of a 1 week period where participants introduce the study product into their diet. Gastrointestinal tolerance, compliance and product acceptability will be evaluated.
Data collection will be performed using paper data collection forms completed by the investigators at the baseline and end of study visits. Patients/parents/guardians, as appropriate, will also complete a brief daily questionnaire on each of the 7 study days. There will also be a questionnaire completed by the patient/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 |
|---|---|---|
| Participant Group | Experimental | Dietary Supplement: PKU EASY Microtabs PlusAll participants will be assessed by their dietitian and prescribed an appropriate amount of the study product, PKU EASY Microtabs Plus, to manage their phenylketonuria. |
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Diagnosis of PKU requiring an appropriate protein substitute
- Subjects who are already taking an appropriate protein substitute and are willing to try the study product for 7 days
- Subjects aged 3 years and above
- Written informed consent obtained from subject or parent/legal guardian, depending on subject age
Key exclusion criteria (5 listed)
- Presence of serious concurrent illness
- Lead Dietitian's uncertainty about the willingness or ability of the patient to comply with the protocol requirements
- Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study, with the exception of sapropterin
- Any subject having taken antibiotics over the previous 2 weeks leading up to the study
- Subjects less than 3 years of age
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 EASY Microtabs PlusPKU EASY Microtabs Plus is a food for special medical purposes (FSMP). This product is for use in the dietary management of Phenylketonuria (PKU) in patients from 3 years of age upwards. PKU EASY Microtabs Plus is a microtablet form, slow-release, phenylalanine-free protein substitute containing a blend of essential and non-essential amino acids, vitamins, minerals and trace elements. | Dietary supplement | Participant Group |
Primary outcome
Change in GI tolerance (participant)
Time frame: Week 0, week 1
Gastrointestinal tolerance assessed using symptom and severity questionnaire completed by the participants. Severity of symptoms will be rated on a scale of 0 - 3, with 0 being no symptoms and 3 being severe or very troublesome symptoms.
Rate of patient compliance with consumption of the prescribed volume of study product
Time frame: Week 1
Assessed via participant diary regarding study product intake. The quantity of study product actually consumed vs the prescribed amount will be recorded. The reason for not consuming the full dose will be recorded by the patient/parent/guardian as free text.
Change in product acceptability
Time frame: Week 1
Assessed via a product acceptability questionnaire completed by the participant/parent/guardian. Organoleptic properties, such as taste and texture, of the study product will be assessed on a rating scale of terrible to great.
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 26 Nov 2025.
- Individual participant dataSharing planned: No. Due to the very small number of participants and rarity of the condition being tested the ability to maintain participant pseudonymity could be compromised.
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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, Ph.D. principal investigator
Birmingham Women's and Children's NHS Foundation Trust
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing PKU EASY Microtabs Plus in people with Phenylketonuria (PKU), run by Galen Limited at 1 site 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 4 inclusion and 5 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 26 Nov 2025. 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 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 GI tolerance (participant) (time frame: Week 0, week 1). The study lists 3 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 26 Nov 2025.