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.
Aperçu de l'étude
Statut
Les conditions
Intervention / Traitement
Type d'étude
Inscription (Estimé)
Phase
- N'est pas applicable
Contacts et emplacements
Coordonnées de l'étude
- Nom: Ellen Taylor
- Numéro de téléphone: +447900724091
- E-mail: ellen.taylor@nutricia.com
Lieux d'étude
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Bristol, Royaume-Uni
- University Hospitals Bristol and Weston NHS Foundation Trust
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Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
- Enfant
- Adulte
- Adulte plus âgé
Accepte les volontaires sains
La description
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
Plan d'étude
Comment l'étude est-elle conçue ?
Détails de conception
- Objectif principal: Science basique
- Répartition: N / A
- Modèle interventionnel: Affectation à un seul groupe
- Masquage: Aucun (étiquette ouverte)
Armes et Interventions
Groupe de participants / Bras |
Intervention / Traitement |
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Expérimental: PKU Microtabet intervention product
Dietary Supplement: PKU Microtablet intervention product
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PKU Microtablet intervention product
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Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
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Gastro-intetsinal tolerance
Délai: 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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Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
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Conformité
Délai: Mesuré au départ (jour 1) et fin de l'intervention (jour 29)
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Le respect de l'apport recommandé du substitut des protéines habituels des patients (pendant la ligne de base) et du produit de l'étude (pendant la période d'étude de cas) sera évalué par la diététiste au départ (jour 1) et à la fin de l'étude de cas (jour 29).
Les patients et / ou les parents / soignants seront invités à rappeler la quantité de leur substitut protéique prise au cours des dernières 24 heures et en moyenne.
Cela sera comparé au montant recommandé pour être consommé par leur diététiste.
Le montant quotidien prescrit par la diététiste gérant les soins du patient sera enregistré au début de l'étude de cas et toute modification de cette ordonnance au cours de l'étude de cas sera également notée.
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Mesuré au départ (jour 1) et fin de l'intervention (jour 29)
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Acceptability
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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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Collaborateurs et enquêteurs
Parrainer
Les enquêteurs
- Directeur d'études: Gary Hubbard, Dr, Nutricia, Inc.
Dates d'enregistrement des études
Dates principales de l'étude
Début de l'étude (Estimé)
Achèvement primaire (Estimé)
Achèvement de l'étude (Estimé)
Dates d'inscription aux études
Première soumission
Première soumission répondant aux critères de contrôle qualité
Première publication (Réel)
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
Dernière mise à jour soumise répondant aux critères de contrôle qualité
Dernière vérification
Plus d'information
Termes liés à cette étude
Termes MeSH pertinents supplémentaires
- Maladies du cerveau
- Maladies du système nerveux central
- Maladies du système nerveux
- Métabolisme, erreurs innées
- Maladies génétiques, innées
- Maladies métaboliques
- Maladies cérébrales, métaboliques, innées
- Maladies cérébrales métaboliques
- Métabolisme des acides aminés, erreurs innées
- Maladies et anomalies congénitales, héréditaires et néonatales
- Maladies nutritionnelles et métaboliques
- Phénylcétonuries
Autres numéros d'identification d'étude
- PKUM2025
Plan pour les données individuelles des participants (IPD)
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Informations sur les médicaments et les dispositifs, documents d'étude
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