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Набор скоро начнётся NCT07671859

PKU Microtablets Case Studies

Без фазы С лечением Phenylketonuria (PKU)

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают
В протоколе указаны: PKU Microtablet protein substitute.
Кому может быть актуально
Состояния в реестре: Phenylketonuria (PKU). Базовые параметры: от 4 лет · Все.
Что важно проверить
Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится
Великобритания
Следующий шаг
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Официальное название

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

Обзор

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.

Вмешательства

  • Пищевая добавка PKU Microtablet protein substitute
    PKU Microtablet intervention product

Первичные конечные точки

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

Критерии участия

Критерии включения

  • 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

Критерии исключения

  • 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение
Не применимо
Модель
Одна группа
Маскирование
Открытое
Основная цель
Фундаментальное исследование

Центры проведения

Великобритания · 1 центр
  • University Hospitals Bristol and Weston NHS Foundation Trust — Bristol

Идентификаторы

NCT: NCT07671859 · PKUM2025

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗