ClinicalTrials.gov record NCT07671859

PKU Microtablets Case Studies

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 6, 2026

Evaluating the Adherence, Tolerance, Acceptability and Safety of a New Amino Acid Based, Microtablet Protein Substitute in PKU: a Case Study Series

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
Age4 years old and older
SexAll
LocationUnited Kingdom
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This interventional case‑study series evaluates a new phenylalanine‑free tablet protein substitute (PKU Microtablets) in people with PKU, including children aged 4 years and older and people. Each case lasts 29 days: a 1‑day baseline followed by a 28‑day period using the study product. Dietitians record symptoms, acceptability, compliance, two finger‑prick blood samples, anthropometry, and adverse events.

What it is studying. The study tests adherence, gastrointestinal tolerance, acceptability, compliance, and safety of PKU Microtablets when taken as a protein substitute alongside a prescribed low‑phenylalanine diet.

What the researchers hope to learn. To evaluate gastrointestinal tolerance, acceptability (sensory experience and ease of use), compliance with the product, and safety (adverse events and metabolic control) of the PKU Microtablet protein substitute.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

Phenylalanine (Phe) free protein substitutes are typically presented in ready to drink liquid or powder format and are made up with water to a set volume. Despite recent advancements related to the taste, scent and texture of commercially available protein substitutes, a proportion of PKU patients choose to consume tablet-based protein substitutes. Protein substitutes in tablet format may help overcome taste, scent and texture concerns associated with powdered and liquid preparations. Indeed, previous research has shown that protein substitute tablets are an effective strategy to manage PKU in children, teenagers and adults. Nonetheless, the daily burden and discipline required often impedes compliance, especially alongside the restrictive low-protein diet. Achieving compliance with multivitamin supplements when given alongside protein substitutes therefore becomes especially challenging. This is particularly true for adolescent, adult and maternal populations. PKU Microtablets have recently been developed. PKU Microtablets are a new phenylalanine-free tablet protein substitute combined with vitamins, minerals and trace elements. As patient acceptability is fundamental to the successful use of any protein substitute, investigation is needed to assess the compliance, tolerance and acceptability of PKU Microtablets as part of a low phenylalanine dietary regimen in PKU patients. This series of case-studies aims to evaluate the gastrointestinal tolerance, acceptability, compliance, and safety of this protein substitute in both adults and children from 4 years. These case studies will last 29 days in total, including a 1-day baseline period followed by a 28-day intervention period. The case studies will be conducted across multiple specialist metabolic centres in the UK.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Dietary supplement
  • PKU Microtablet protein substitute

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • Male or female
  • Over 4 years of age
  • Patients diagnosed with any phenotype of PKU characterised by hyperphenylalaninaemia
  • Have been compliant in taking at least one protein substitute, providing at least 20g protein equivalent, for at least 1 month prior to trial commencement
  • Have a prescribed daily phenylalanine allowance for PKU
  • Written or electronic informed consent from patient, and/or from parent/caregiver if applicable
  • Participants who are anticipated to be able to take at least 20g protein equivalent of the case study product per day

Exclusion criteria

  • Pregnant or lactating
  • Requiring parenteral nutrition
  • Major hepatic or renal dysfunction
  • Participation in other studies within 1 month prior to entry of this study
  • Allergy to any of the study product ingredients
  • Investigator concern around willingness/ability of patient or parent/caregiver to comply with protocol requirement
  • Treatment with Sapropterin dihydrochloride for less than 6 months

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

The case study lasts 29 days (1-day baseline, then 28 days on the study product); participants will take the PKU Microtablet protein substitute daily. A dietitian will record gastrointestinal symptoms and product acceptability at baseline (Day 1) and at the end (Day 29), measure height (baseline) and weight (baseline and Day 29), and record medical and treatment history. Participants will provide two fasting finger‑prick dried blood spot samples (one at the start of the intervention period before taking the product, and one at Day 29) for blood phenylalanine and tyrosine testing. Adverse events will be recorded throughout, and the dietitian will assess compliance by asking the participant or caregiver about recent intake.

How the study is structured. Interventional, single‑group case‑study series with no masking. Participants have a 1‑day baseline then take the PKU Microtablet product for 28 days. Dietitians collect symptom questionnaires, acceptability ratings, recall of intake, two fasting dried blood‑spot samples, height/weight, and record adverse events.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The registry says recruitment has not started. When is it expected to open, and can I register interest now?
  • The listed sites are in United Kingdom. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on phenylketonuria

Clinical trials38 trials listed for phenylketonuriaEvery trial the registry lists for phenylketonuria, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in phenylketonuria researchLatest monthly update: September 2026. Includes: Diet supply gaps. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Oct 1, 2026 (estimated)
Primary completion
Jun 1, 2027 (estimated)
Study completion
Jul 1, 2027 (estimated)
Target enrollment
20 participants (estimated)
Last updated on registry
Sep 23, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Nutricia UK Ltd

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

  • University Hospitals Bristol and Weston NHS Foundation Trust

    Bristol · United Kingdom

Terms used on this page · explanations by MedicalUpdates.ai

baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

← Back to clinical trials