Market research - acceptability study for a new PKU protein substitute

  • Research type

    Research Study

  • Full title

    Market research - acceptability study for a new PKU glycomacropeptide based protein substitute (Dr Schär- Mevalia Amino Acids) Mevalia PKU GMPower - vanilla

  • IRAS ID

    256336

  • Contact name

    Anita MacDonald

  • Contact email

    Anita.Macdonald@nhs.net

  • Sponsor organisation

    Dr. Schär

  • Clinicaltrials.gov Identifier

    n/a, n/a

  • Duration of Study in the UK

    0 years, 1 months, 12 days

  • Research summary

    This is an acceptability study for a new PKU glycomacropeptide based protein substitute product. Protein substitutes are an integral part of the dietary management of Phenylketonuria (PKU), an inborn error of metabolism. This study seeks to ascertain whether GMPower is palatable for patients, and whether it is tolerated by patients e.g. whether there are any side effects such as vomiting, tummy aches or diarrhoea (common side effects in products of this nature, already Advisory Committee on Borderline Substances (ACBS) approved). We are also looking to include whether it is practical for daily use, for example, how easy/difficult are it is to consume and administer. Participants from 3 years of age to adulthood will be included in the study. The product is suitable for this age range. The outcome of this market research will be included in our application to the ACBS in order to gain approval for use on prescription. An acceptability study is an obligatory part of the ACBS application. The results of this study will not be published or used in any medical texts. This is a market research exercise for our products, which are already available and tolerated in patients with PKU in other European countries (Germany and Italy). The composition of the new product is comparable to similar products currently available on the UK market, which are also approved by the ACBS. Long-term compliance with taking protein substitutes is an ongoing challenge in these patients and as a low phenylalanine diet is recommended for life, improving the choice in terms of new products may aid compliance.
    Participants will be asked to take the new product for 7 days. During this time parents/caregivers or the patient (where appropriate) will be asked to complete a daily questionnaire recording information on:
    • Volume of product taken
    • Ease and/or 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 questionnaire should take a maximum of 30 minutes to complete.

  • REC name

    North West - Liverpool Central Research Ethics Committee

  • REC reference

    18/NW/0828

  • Date of REC Opinion

    3 Jan 2019

  • REC opinion

    Further Information Favourable Opinion