Evaluation of PKU UP

• Research type

  Research Study

• Full title

  An acceptability study to evaluate the compliance, gastrointestinal tolerance, palatability and metabolic control of children with phenylketonuria (PKU) when using PKU UP (a food for special medical purposes) as part of dietary management.

• IRAS ID

  327641

• Contact name

  Anita MacDonald

• Contact email

  Anita.MacDonald@BCH.NHS.UK

• Sponsor organisation

  Vitaflo (International) Ltd

• Clinicaltrials.gov Identifier

  NCT05995717

• Duration of Study in the UK

  1 years, 6 months, 0 days

• Research summary

  We propose to recruit children with a diagnosis of phenylketonuria (PKU) aged 1-10 years. PKU is a rare inborn error of metabolism with a prevalence of 1 in 10,000 in the UK population. The mainstay of treatment for most individuals is dietary therapy with a strict protein-restricted diet.

  The study product, PKU UP, is a food for special medical purposes (FSMP), as defined by the Delegated Act EU 2016/128, used for the dietary management of Phenylketonuria (PKU).

  The study will be looking at the acceptability of PKU UP, as defined by the Advisory Committee on Borderline Substances (ACBS). This includes the following:
  • participant adherence to recommended intakes
  • gastrointestinal symptoms
  • evaluations of palatability.

  Each participant will be on the study for 26 weeks. This includes evaluation part 1 and evaluation part 2.
  Part 1 consists of a 12 week period where participants introduce the dietary therapy into their diet. Gastrointestinal tolerance, metabolic control, compliance, product acceptability and dietary quality will be evaluated.
  Part 2 consists of a 14 week period where participants continue to take the dietary therapy for a longer assessment of growth and nutritional status.

  Data collection will be performed using paper and/or electronic Case Report Forms completed by the investigators at the baseline, evaluation periods and end of study visits. There will also be three questionnaires completed by the parents/guardians over the course of the study, in relation to protein substitute intake, gastrointestinal tolerance, and product acceptability.

  Pending the outcome of the study, Vitaflo intends to submit an application to the ACBS for PKU UP to be made available via prescription.

• REC name

  North West - Liverpool Central Research Ethics Committee

• REC reference

  23/NW/0185

• Date of REC Opinion

  11 Jul 2023

• REC opinion

  Favourable Opinion