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.
Panoramica dello studio
Stato
Condizioni
Intervento / Trattamento
Tipo di studio
Iscrizione (Stimato)
Fase
- Non applicabile
Contatti e Sedi
Contatto studio
- Nome: Ellen Taylor
- Numero di telefono: +447900724091
- Email: ellen.taylor@nutricia.com
Luoghi di studio
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Bristol, Regno Unito
- University Hospitals Bristol and Weston NHS Foundation Trust
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Criteri di partecipazione
Criteri di ammissibilità
Età idonea allo studio
- Bambino
- Adulto
- Adulto più anziano
Accetta volontari sani
Descrizione
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
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Scienza basilare
- Assegnazione: N / A
- Modello interventistico: Assegnazione di gruppo singolo
- Mascheramento: Nessuno (etichetta aperta)
Armi e interventi
Gruppo di partecipanti / Arm |
Intervento / Trattamento |
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Sperimentale: PKU Microtabet intervention product
Dietary Supplement: PKU Microtablet intervention product
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PKU Microtablet intervention product
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Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Gastro-intetsinal tolerance
Lasso di tempo: 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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Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Conformità
Lasso di tempo: Misurato al basale (giorno 1) e alla fine dell'intervento (giorno 29)
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La conformità con l'assunzione raccomandata del solito sostituto della proteina dei pazienti (durante il basale) e con il prodotto di studio (durante il periodo di case study) sarà valutata dal dietista al basale (giorno 1) e alla fine del case study (giorno 29).
Ai pazienti e/o genitori/caregiver verrà chiesto di ricordare la quantità del loro sostituto delle proteine assunto nelle ultime 24 ore e in media.
Ciò sarà confrontato con la quantità raccomandata per essere consumata dal dietista.
L'importo giornaliero prescritto dal dietista che gestisce le cure del paziente sarà registrata all'inizio del case study e verranno anche annotate eventuali modifiche a questa prescrizione durante il case study.
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Misurato al basale (giorno 1) e alla fine dell'intervento (giorno 29)
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Acceptability
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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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Collaboratori e investigatori
Sponsor
Investigatori
- Direttore dello studio: Gary Hubbard, Dr, Nutricia, Inc.
Studiare le date dei record
Studia le date principali
Inizio studio (Stimato)
Completamento primario (Stimato)
Completamento dello studio (Stimato)
Date di iscrizione allo studio
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Effettivo)
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo verificato
Maggiori informazioni
Termini relativi a questo studio
Termini MeSH pertinenti aggiuntivi
- Malattie del cervello
- Malattie del sistema nervoso centrale
- Malattie del sistema nervoso
- Metabolismo, errori congeniti
- Malattie genetiche, congenite
- Malattie metaboliche
- Malattie cerebrali, metaboliche, congenite
- Malattie cerebrali, metaboliche
- Metabolismo degli aminoacidi, errori congeniti
- Malattie e anomalie congenite, ereditarie e neonatali
- Malattie nutrizionali e metaboliche
- Fenilchetonuria
Altri numeri di identificazione dello studio
- PKUM2025
Piano per i dati dei singoli partecipanti (IPD)
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Informazioni su farmaci e dispositivi, documenti di studio
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