Evaluation of PKU Start

Not Applicable

Completed

• Conditions: Phenylketonuria; Inborn Errors of Metabolism
• Interventions: Dietary Supplement: PKU Start

• Registration Number: NCT03058848
• Lead Sponsor: Vitaflo International, Ltd

Brief Summary

To evaluate the acceptability, tolerance and effect on metabolic control of PKU Start, a new Phe free protein substitute for the dietary management of PKU in infants from birth.

Detailed Description

This is an assessment of ten (10) infants who require a protein restricted diet that is low in Phe. Infants who routinely use a Phe free infant formula as part of their dietary therapy will be recruited for a 28-day assessment of PKU Start, to evaluate tolerance and acceptability.

The outcome of this assessment will be used in a submission to the regulatory authorities, Advisory Committee on Borderline Substances (ACBS), for PKU Start to become reimbursable on prescription in the UK.

The participant's dietitian will advise on an appropriate amount of PKU Start based on individual requirements. Parents/carers will be asked to substitute their usual Phe-free infant formula with PKU Start for one (1) month. The sponsor will supply PKU Start free of charge.

Prior to starting PKU Start, parents/carers will be asked to record information about the infant's usual GI tolerance and feeding pattern for a period of three (3) days, to allow for comparison between their existing formula and PKU Start.

They will be asked to record information about the following:

Stools Vomiting and Spit-up Feed / Fluid Intake and Compliance Phenylalanine Levels Final Evaluation about the presentation of the product, ease of preparation and how PKU Start flowed through the teat of a bottle.

Study Timeline

• Study First Submitted: 2017-02-16
• Study First Submitted QC: 2017-02-16
• Study First Posted: 2017-02-23
• Study Start: 2017-03-06
• Primary Completion: 2017-09-15
• Study Completion: 2017-09-15
• Status Verified: 2024-02
• Last Update Submitted: 2024-02-15
• Last Update Posted: 2024-02-16

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

1. A diagnosis of classical or severe PKU on new-born screening (For the purposes of this study, 'severe' is defined as phe concentrations between 120 and 600 µmol/L at diagnosis)
2. Taking a minimum of one (1) feed of a Phe-free infant formula
3. A minimum period of four (4) weeks from the time of diagnosis to initial approach to parents

Exclusion Criteria

1. Diagnosed with mild PKU or hyperphenylalaninaemia (For the purposes of this study, 'mild' is defined as phe concentrations between 120 and 600 µmol/L at diagnosis)
2. Diagnosis of a congenital condition

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Consumption of PKU Start	PKU Start	Daily feed, substituting the participant's normal phe-free formula for PKU Start.

Primary Outcome Measures

Name	Time	Method
Daily phenylalanine control	Days 1-28	Collection of quantitative data regarding phenylalanine control using routine biochemical testing
GI tolerance daily diary	Days 1-28	Qualitative assessments from subject questionnaires that allow evaluation of the gastro-intestinal tolerance of the study product.
Product compliance daily diary	Days 1-28	Quantitative assessments from subject questionnaires that allow evaluation of compliance with the study product, i.e. actual versus prescribed intake.
Ease of use questionnaire	Day 29	Qualitative assessment from subject questionnaire that allows evaluation of the ease of use of the study product.

Trial Locations

Locations (3)

Birmingham Children's Hospital NHS Foundation Trust; 🇬🇧 Birmingham, West Midlands, United Kingdom

NHS Greater Glasgow and Clyde; 🇬🇧 Glasgow, Lanarkshire, United Kingdom

Bradford Teaching Hospitals NHS Foundation Trust; 🇬🇧 Bradford, West Yorkshire, United Kingdom