PKU GOLIKE PLUS 16+ - Market research study - version 1

• Research type

  Research Study

• Full title

  PKU GOLIKE PLUS 16+ - Acceptability and tolerance market research study

• IRAS ID

  265934

• Contact name

  Charlotte Ellerton

• Contact email

  c.ellerton@nhs.net

• Sponsor organisation

  APR Applied Pharma Research SA

• Duration of Study in the UK

  0 years, 3 months, 13 days

• Research summary

  The main treatment for patients 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 16+ (APR Applied Pharma Research S.A.) is a protein substitute designed for patients with PKU, aged ≥16 years.

  This prospective market research study aims at evaluating the acceptability and gastrointestinal tolerance of this new protein substitute in 15 patients with PKU, aged ≥16 years.
  Subjects who are currently taking a protein substitute for PKU will be recruited for a 7-day trial on the new protein substitute.
  The treatment with the new protein substitute will last for 7 days. PKU GOLIKE PLUS 16+ could replace partially or totally the amount of the previous protein substitute/s.

  During the 7-day treatment at home, patients will be asked to fill in a daily diary by recording information on:
  • intake of the product (actual vs prescribed; how it is taken) and compliance;
  • any gastrointestinal signs/symptoms.
  In addition, patients will be asked to fill in an “Acceptability Questionnaire” in which to record any perception about visual appearance, smell, taste, after-taste, palatability, and overall liking of the product/potential interest to take it on a long-term basis.
  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

  London - Camden & Kings Cross Research Ethics Committee

• REC reference

  19/LO/1610

• Date of REC Opinion

  22 Oct 2019

• REC opinion

  Favourable Opinion