Evaluation of PKU UP

PKU Clinical Trial

— PKU UP  

Official title:

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.

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05995717
• Other study ID #: PKU-PAED-LIQ-2022-10-07
• Secondary ID: 32764123/NW/0185
• Status: Recruiting
• Phase: N/A
• Start date: January 11, 2024
• Est. completion date: September 11, 2025

Study information

• Verified date: February 2024
• Source: Vitaflo International, Ltd
• Contact: Clinical Trials
• Phone: 0151 709 9020
• Email: ClinicalTrialsTeam[@]Vitaflo.co.uk
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 15
• Est. completion date: September 11, 2025
• Est. primary completion date: July 11, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 10 Years

Eligibility

Inclusion Criteria:

• 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 are anticipated to be able to take at least half of their daily protein equivalent requirement from PKU UP.

• Willingly given, written, informed consent from the parent(s)/guardian(s).

• Willingly given, written assent by the participant (if appropriate).

Exclusion Criteria:

• 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).

Related Conditions & MeSH terms

• Phenylketonurias
• PKU

Intervention

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).

Locations

Country	Name	City	State
United Kingdom	Royal Belfast Hospital for Sick Children	Belfast
United Kingdom	Birmingham Children's Hospital	Birmingham
United Kingdom	University Hospitals Bristol and Weston NHS Foundation Trust	Bristol
United Kingdom	Great Ormond Street Hospital for Children NHS Foundation Trust	London

Sponsors (2)

Lead Sponsor	Collaborator
Vitaflo International, Ltd	Birmingham Women's and Children's NHS Foundation Trust

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in phe level between baseline and visit 3	Change in phe level as measured by dried blood spot testing	26 weeks
Primary	Change in tyrosine level between baseline and visit 3	Change in tyrosine level as measured by dried blood spot testing	26 weeks
Primary	Change in PI impression of metabolic control	Assessment of the participant's metabolic control in the opinion of the investigator	Week 0, week 6, week 12, week 26
Primary	Change in the growth of participants as measured by Height (length in under 2 years of age), Head circumference in under 2 years of age and Weight.	Assessed via anthropometry measurements. Participants will be weighed and measured for growth monitoring.	Week 0, week 12, week 26
Primary	Change in GI tolerance (participant)	Gastrointestinal tolerance assessed using the PedsQL Gastrointestinal Symptoms Scale completed by the participants	Week 0, week 6, week 12, week 26
Primary	Change in PI impression of GI tolerance	Assessment of the participant's GI tolerance in the opinion of the investigator	Week 0, week 6, week 12, week 26
Primary	Change in compliance with study product consumption	Assessed via participant diary regarding study product intake	Week 0, week 6, week 12, week 26
Primary	Change in PI impression of study product intake	Assessment of the participant's compliance in the opinion of the investigator	Week 0, week 6, week 12, week 26
Primary	Change in product acceptability	Assessed via a product acceptability questionnaire completed by the participant/parent	Week 0, week 12, week 26
Primary	Change in dietary intake	Assessed via 24-hour recall and low protein food frequency questionnaire	Week 0, week 12, week 26
Primary	Change in nutritional biochemistry	Assessed via venous blood sample of approximately 7ml	Week 0, week 26