TYR EASY Tablets - ACCEPTABILITY AND TOLERANCE
Research type
Research Study
Full title
TYR EASY Tablets - ACCEPTABILITY AND TOLERANCE
IRAS ID
272118
Contact name
Anita MacDonald
Contact email
Sponsor organisation
Galen Ltd
Duration of Study in the UK
0 years, 3 months, 25 days
Research summary
Summary of Research
The principle treatment for children with Tyrosinaemia (TYR) 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.
This is a prospective, observational market research study in 8 children with TYR to evaluate the acceptability, tolerance and compliance with TYR EASY Tablets, a protein substitute for use in the dietary management of patients with TYR from 8 years and above.
Subjects who are currently taking a protein substitute for TYR will be recruited for a 28-day trial of the new protein substitute to evaluate the tolerability and acceptability of the study product.
During the 28-day trial patients or caregivers(depending on ability) 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 end of the study that will consider perceptions about taste, appearance, smell, presentation and packaging of the product; ease of administration; how it is taken; and any other problems or symptoms.
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.Summary of Results
This is the first tablet to be used as a protein substitute in tyrosinemia. Despite the large number of tablets that need to be taken each day the subject group who took the tablets overwhelmingly preferred them to a liquid or spoonable protein substitute. In the 5 children who completed the 28 day study, all of them had improved metabolic control with 100% (n=5) having a lower tyrosine concentration. When compared with the previous 12 months 60% (n=3) compared to 20% (n=1) met the recommended target tyrosine concentrations (200-400μmol/L).
REC name
HSC REC B
REC reference
19/NI/0219
Date of REC Opinion
8 Jan 2020
REC opinion
Further Information Favourable Opinion