PKU GOLIKE PLUS 3-16 - MARKET RESEARCH STUDY - Version 1
Research type
Research Study
Full title
PKU GOLIKE PLUS 3-16 - Acceptability and tolerance market research study
IRAS ID
256519
Contact name
Anita MacDonald
Contact email
Sponsor organisation
APR Applied Pharma Research SA
ISRCTN Number
ISRCTN12387189
Duration of Study in the UK
0 years, 4 months, 29 days
Research summary
The principle treatment for children with phenylketonuria (PKU) is a low protein diet. Part of this treatment requires the administration of a protein substitute. Several brands of protein substitutes are already available in the UK in various presentations. However, compliance with taking protein substitutes continues to be a challenge. As a low protein diet is recommended for life, long-term compliance is a growing concern. As a result, improving the choice in terms of product type may aid compliance.
PKU GOLIKE PLUS 3-16 (APR Applied Pharma Research S.A.) is a protein substitute designed for children with PKU, aged 3-16 years.
This prospective, observational market research study aims at evaluating the acceptability and tolerability of this new protein substitute in 8-10 children with PKU, aged 3-16 years.
Subjects who are currently taking a protein substitute for PKU will be recruited for a 7-day trial of the new protein substitute. The treatment with the new protein substitute will last for 7 days.During the 7-day trial, caregivers will be asked to complete a daily questionnaire recording information on:
• Usage and compliance;
• Ease of use and any issues with administration;
• Any gastro-intestinal side-effects.A questionnaire will also be completed at the beginning and at the end of the study and it will consider perceptions about taste, appearance, smell, presentation and packaging of the new product; ease of administration; how it is taken; and any other problems or symptoms.
Finally, subjects will be asked to undergo routine biochemical testing, in fasting conditions, at the beginning and at the end of the trial, for measuring blood phenylalanine levels.
The outcome of this assessment will be used in a submission to regulatory authorities to get the study product reimbursable on prescription in the UK.
REC name
West Midlands - Solihull Research Ethics Committee
REC reference
19/WM/0151
Date of REC Opinion
25 Jun 2019
REC opinion
Further Information Favourable Opinion