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Not yet recruiting NCT07671859

PKU Microtablets Case Studies

No phase Interventional Phenylketonuria (PKU)

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: PKU Microtablet protein substitute.
Who it may be relevant to
Registry conditions: Phenylketonuria (PKU). Basic parameters: from 4 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United Kingdom
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

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Official title

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

Overview

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

  • Dietary supplement PKU Microtablet protein substitute
    PKU Microtablet intervention product

Primary outcome measures

  • Gastro-intetsinal tolerance [Time frame: Measured at baseline (day 1) and end of intervention (day 29)]
Secondary outcome measures (7)
  • Acceptability [Time frame: Measured at baseline (day 1) and end of intervention (day 29)]
  • Compliance [Time frame: Measured at baseline (day 1) and end of intervention (day 29)]
  • Blood Phe and Tyrosine [Time frame: Day 2 (baseline) and day 29 (end of intervention)]
  • Anthropometry - Height [Time frame: Day 1 (baseline) and day 29 (end of intervention)]
  • Anthropometry - Weight [Time frame: Day 1 (baseline) and day 29 (end of intervention)]
  • Patient History [Time frame: Day 1 (baseline) and day 29 (end of intervention)]
  • Safety/ Adverse events [Time frame: Throughout the case study series (day 1 to day 29).]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Basic science

Study locations

United Kingdom · 1 center
  • University Hospitals Bristol and Weston NHS Foundation Trust — Bristol

Identifiers

NCT: NCT07671859 · PKUM2025

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗