PKU Microtablets Case Studies
Evaluating the Adherence, Tolerance, Acceptability and Safety of a New Amino Acid Based, Microtablet Protein Substitute in PKU: a Case Study Series
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.
Studieöversikt
Status
Betingelser
Intervention / Behandling
Studietyp
Inskrivning (Beräknad)
Fas
- Inte tillämpbar
Kontakter och platser
Studiekontakt
- Namn: Ellen Taylor
- Telefonnummer: +447900724091
- E-post: ellen.taylor@nutricia.com
Studieorter
-
-
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Bristol, Storbritannien
- University Hospitals Bristol and Weston NHS Foundation Trust
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Deltagandekriterier
Urvalskriterier
Åldrar som är berättigade till studier
- Barn
- Vuxen
- Äldre vuxen
Tar emot friska volontärer
Beskrivning
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
Studieplan
Hur är studien utformad?
Designdetaljer
- Primärt syfte: Grundläggande vetenskap
- Tilldelning: N/A
- Interventionsmodell: Enskild gruppuppgift
- Maskning: Ingen (Open Label)
Vapen och interventioner
Deltagargrupp / Arm |
Intervention / Behandling |
|---|---|
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Experimentell: PKU Microtabet intervention product
Dietary Supplement: PKU Microtablet intervention product
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PKU Microtablet intervention product
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Vad mäter studien?
Primära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
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Gastro-intetsinal tolerance
Tidsram: Measured at baseline (day 1) and end of intervention (day 29)
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GI tolerance will be recorded by the Dietitian at baseline on Day 1 and at the end of the case study period (Day 29).
GI tolerance will be recorded using a standardised questionnaire to capture perceived severity (none, mild, moderate or severe) of common gastrointestinal symptoms (diarrhoea, constipation, bloating, abdominal discomfort, vomiting and nausea).
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Measured at baseline (day 1) and end of intervention (day 29)
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Sekundära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
|
Efterlevnad
Tidsram: Mätt vid baslinjen (dag 1) och slutet av interventionen (dag 29)
|
Överensstämmelse med det rekommenderade intaget av patienternas vanliga proteinsubstitut (under baslinjen) och med studieprodukten (under fallstudieperioden) kommer att utvärderas av dietisten vid baslinjen (dag 1) och i slutet av fallstudien (dag 29).
Patienter och/eller föräldrar/vårdgivare kommer att uppmanas att återkalla mängden av deras proteinsubstitut som tagits under det senaste dygnet och i genomsnitt.
Detta kommer att jämföras med det belopp som rekommenderas att konsumeras av deras dietist.
Det dagliga beloppet som föreskrivs av dietisten som hanterar patientens vård kommer att registreras i början av fallstudien och eventuella ändringar av detta recept under fallstudien kommer också att noteras.
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Mätt vid baslinjen (dag 1) och slutet av interventionen (dag 29)
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Acceptability
Tidsram: Measured at baseline (day 1) and end of intervention (day 29)
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Acceptability (ease of use and liking) of the patients' usual protein substitute and the study product will be assessed by the Dietitian at baseline (Day 1) and the end of the case study period (Day 29) by a series of questions posed to the patient and/or parent/caregiver.
Acceptability questions will assess the patient's sensory experience, ease of use, tolerance, and overall self-rated acceptability on a 5-point Likert scale from Terrible (worst) to Great (best).
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Measured at baseline (day 1) and end of intervention (day 29)
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Blood Phe and Tyrosine
Tidsram: Day 2 (baseline) and day 29 (end of intervention)
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Participants will be required to provide a total of two fasting fingerprick blood samples as dried blood spots; one on the first day of the intervention period (Day 2) before consuming any of the case study product, and one at the end of the intervention period (Day 29).
These tests will be conducted in addition to any usual blood tests the participants conduct as part of their usual disease management, which vary in frequency with the age of the participant and the local policy.
Dietitians will also be asked to describe the patient's metabolic control during the case study period.
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Day 2 (baseline) and day 29 (end of intervention)
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Anthropometry - Height
Tidsram: Day 1 (baseline) and day 29 (end of intervention)
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Height will be measured, using standard methods, recorded to the nearest 0.1cm (at baseline only) and used to calculate body mass index (BMI, kg/m2).
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Day 1 (baseline) and day 29 (end of intervention)
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Anthropometry - Weight
Tidsram: Day 1 (baseline) and day 29 (end of intervention)
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For safety purposes, at baseline (Day 1) and at the end of the case study (Day 29), body weight (kg) will be measured where possible using standard methods, recorded to the nearest 0.1kg using a weighing scale without heavy clothing.
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Day 1 (baseline) and day 29 (end of intervention)
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Patient History
Tidsram: Day 1 (baseline) and day 29 (end of intervention)
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A detailed patient history will be recorded at baseline to assess previous compliance to protein substitutes, relevant medical and clinical issues, medication(s) prescribed, and any other relevant information related to the patient's condition or dietary management.
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Day 1 (baseline) and day 29 (end of intervention)
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Safety/ Adverse events
Tidsram: Throughout the case study series (day 1 to day 29).
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All adverse events will be recorded, throughout the case study.
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Throughout the case study series (day 1 to day 29).
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Samarbetspartners och utredare
Sponsor
Utredare
- Studierektor: Gary Hubbard, Dr, Nutricia, Inc.
Studieavstämningsdatum
Studera stora datum
Studiestart (Beräknad)
Primärt slutförande (Beräknad)
Avslutad studie (Beräknad)
Studieregistreringsdatum
Först inskickad
Först inskickad som uppfyllde QC-kriterierna
Första postat (Faktisk)
Uppdateringar av studier
Senaste uppdatering publicerad (Faktisk)
Senaste inskickade uppdateringen som uppfyllde QC-kriterierna
Senast verifierad
Mer information
Termer relaterade till denna studie
Ytterligare relevanta MeSH-villkor
- Hjärnsjukdomar
- Sjukdomar i centrala nervsystemet
- Sjukdomar i nervsystemet
- Metabolism, medfödda fel
- Genetiska sjukdomar, medfödda
- Metaboliska sjukdomar
- Hjärnsjukdomar, metabola, medfödda
- Hjärnsjukdomar, metaboliska
- Aminosyrametabolism, medfödda fel
- Medfödda, ärftliga och neonatala sjukdomar och abnormiteter
- Närings- och metabola sjukdomar
- Fenylketonuri
Andra studie-ID-nummer
- PKUM2025
Plan för individuella deltagardata (IPD)
Planerar du att dela individuella deltagardata (IPD)?
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