NCT05995717

Evaluation of PKU UP

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.

• Status: Active Not Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 16 (actual)
• Sponsor: Vitaflo International, Ltd · Industry
• Sex: All
• Age: 1 Year – 10 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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: * Part 1 consists of a 12 week period where participants introduce the study product into their diet. Gastrointestinal tolerance, metabolic control, compliance, product acceptability and dietary quality will be evaluated. * Part 2 consists of a 14 week period where participants continue to take the study product for a longer assessment of growth and nutritional status. Data collection will be performed using paper and/or electronic Case Report Forms completed by the investigators at the baseline, evaluation periods and end of study visits. There will also be three questionnaires completed by the parents/guardians over the course of the study, in relation to protein substitute intake, gastrointestinal tolerance, and product acceptability.

Conditions

• PKU

Interventions

Type	Name	Description
DIETARY_SUPPLEMENT	PKU UP	PKU UP is a food for special medical purposes (FSMP). This product is for use in the dietary management of Phenylketonuria (PKU) in children aged from one (1) to ten (10) years. PKU UP is a neutral-flavoured, ready-to-drink, low-phenylalanine protein substitute containing a blend of glycomacropeptide (GMP) isolate, essential and non-essential amino acids, fat, carbohydrate, vitamins, minerals and docosahexaenoic acid (DHA).

Timeline

• Start date: 2024-01-11
• Primary completion: 2026-05-31
• Completion: 2026-05-31
• First posted: 2023-08-16
• Last updated: 2026-02-19

Locations

7 sites across 1 country: United Kingdom