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Идёт набор NCT06941532

GMP Powdered Substitutes in PKU and TYR

Без фазы С лечением Phenylketonuria Tyrosinemia

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: GMP Intervention Product.
Кому может быть актуально
Состояния в реестре: Phenylketonuria, Tyrosinemia. Базовые параметры: от 3 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 New GMP-based Powdered Protein Substitutes in PKU and TYR: a Case Study Series

Обзор

Four new GMP-based protein substitutes have been developed to support the dietary management of PKU and TYR. These products are powdered protein substitutes, low in phenylalanine and low in phenylalanine and tyrosine respectively, with a mix of glycomacropeptide (GMP), essential and non-essential amino acids, carbohydrates, fibres, fats (including DHA) and micronutrients. The proteins in these new protein substitutes are based on GMP, a peptide isolated from milk during cheese manufacturing and the only known naturally derived protein source low in phenylalanine and tyrosine. The GMP is supplemented with other amino acids which are naturally low or not present in GMP, as well as carbohydrates, fats, fibre and micronutrients. Studies to date have illustrated that PKU patients who replace their regular phenylalanine-free amino acid-based formula with GMP-based foods may have better diet compliance and prefer the taste whilst maintaining metabolic control. This series of case-studies aims to evaluate the gastrointestinal tolerance, acceptability, compliance, and safety of these four GMP based protein substitutes in both adults and children over 3 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. A series of case studies is undertaken due to the rarity of these conditions, the diversity of patient types and the difficulty in recruiting these patients to trials.

Вмешательства

  • Пищевая добавка GMP Intervention Product
    Condition specific GMP based intervention product

Первичные конечные точки

  • Gastro-intetsinal tolerance [Срок оценки: Measured at baseline (day 1) and end of intervention (day 29)]
Вторичные конечные точки (6)
  • 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 phenylalanine, blood tyrosine and other amino acid levels [Срок оценки: Day 1 (baseline) and day 29 (end of intervention)]
  • Anthropometry [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 3 years of age
  • Patients diagnosed with any phenotype of PKU characterised by hyperphenylalaninaemia or patients diagnosed with tyrosinaemia
  • Have been compliant in taking at least one protein substitute, providing at least 15g protein equivalents, for at least 1 month prior to trial commencement
  • Have a prescribed daily phenylalanine or phenylalanine and tyrosine allowance for PKU or TYR respectively
  • 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 one sachet of GMP 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

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
  • Birmingham Women and Children's Hospital — Birmingham

Идентификаторы

NCT: NCT06941532 · GMP2024

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗