An Acceptability Study to Evaluate the Compliance, Gastrointestinal Tolerance, Palatability and Metabolic Control of Children With Phenylketonuria (PKU) When Using PKU UP (a Food for Special Medical Purposes) as Part of Dietary Management.

Vitaflo International, Ltd7 个研究点分布在 1 个国家目标入组 16 人开始时间: 2024年1月11日最近更新: 2026年5月18日

适应症

干预措施

试验速览

阶段: 不适用
状态: 已完成
发起方: Vitaflo International, Ltd
入组人数: 16
试验地点: 7
主要终点: Change in phe level between baseline and visit 3

研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

PKU UP is a prospective, single-arm, open-label, 26-week acceptability study to evaluate PKU UP for the dietary management of participants with phenylketonuria (PKU). Up to 15 participants aged 1 - 10 years old will be recruited and it is anticipated the study will open in at least three sites in the United Kingdom (UK) to recruit the target number of participants in the required timeframe.

详细描述

PKU is a rare inborn error of metabolism with a prevalence of 1 in 10,000 in the UK population. The mainstay of treatment for most individuals is dietary therapy with a strict protein-restricted diet. We propose to recruit children with a diagnosis of PKU aged 1-10 years.

The study product, PKU UP, is a food for special medical purposes (FSMP), as defined by the Delegated Act EU 2016/128, used for the dietary management of Phenylketonuria (PKU).

The study will be investigating the acceptability of PKU UP, as defined by the Advisory Committee on Borderline Substances (ACBS). This includes the following:

participant adherence to recommended intakes
gastrointestinal symptoms
evaluations of palatability.

Each participant will be on the study for 26 weeks. This includes evaluation part 1 and evaluation part 2:

研究设计

研究类型: Interventional
分配方式: Na
干预模型: Single Group
主要目的: Treatment
盲法: None

入排标准

年龄范围: 1 Year 至 10 Years（Child）
性别: All
接受健康志愿者: 否

入选标准

•Diagnosis of PKU requiring a low-protein diet and protein substitute.
•Participants who are adherent with their blood phenylalanine (Phe) testing, in the opinion of the investigator.
•Participants aged 1-10 years.
•Participants who in the opinion of the investigator are anticipated to be able to take at least half of their daily protein equivalent requirement from PKU UP (in order to assess this, a taste test (or similar) may be conducted at investigator's discretion as part of the screening process).
•Willingly given, written, informed consent from the parent(s)/guardian(s).
•Willingly given, written assent by the participant (if appropriate).

排除标准

•Inability to comply with the study protocol, in the opinion of the investigator.
•Any co-morbidity, which, in the opinion of the investigator, would preclude participation in the study.
•Diagnosis of persistent hyperphenylalaninemia, or mild PKU not requiring a low protein diet and protein substitute.
•Allergy or intolerance to milk.
•Participants who are currently participating in, plan to participate in or have participated in an interventional investigational drug, food or medical device study within 30 days prior to the screening visit.
•Use of additional micronutrient supplements during the evaluation period, unless clinically indicated and prescribed by the Investigator (must be recorded in both the medical records and case report form).

研究组 & 干预措施

PKU UP

Experimental

All participants will be assessed by their dietitian and prescribed an appropriate amount of the study product, PKU UP, to manage their phenylketonuria.

干预措施: PKU UP (Dietary Supplement)

结局指标

主要结局

Change in phe level between baseline and visit 3

时间窗: 26 weeks

Change in phe level as measured by dried blood spot testing

Change in tyrosine level between baseline and visit 3

时间窗: 26 weeks

Change in tyrosine level as measured by dried blood spot testing

Change in PI impression of metabolic control