RMD Lophlex Powders Case Studies
Research type
Research Study
Full title
Evaluating the acceptability, compliance and tolerance of the powder formats of three existing protein substitutes for the dietary management of rare metabolic diseases in children and adults – case series
IRAS ID
255107
Contact name
Rebecca Stratton
Contact email
Sponsor organisation
Nutricia Ltd (UK)
Duration of Study in the UK
2 years, 4 months, 30 days
Research summary
These are case studies, not a clinical trial.\n\nHomocystinuria (HCU), Maple Syrup Urine Disease (MSUD) and Tyrosinemia (TYR) type II are rare metabolic disorders (RMDs) of protein metabolism that require removal of specific amino acids from the diet. The dietary management of these conditions requires the consumption of protein substitute(s) free of specific amino acids and a calculated intake of protein containing foods to keep the blood amino acid concentrations within the desired ranges. \n\nIt is widely accepted that having a variety of protein substitutes available for patients may improve compliance. \n\nAs such, Nutricia Advanced Medical Nutrition has developed the protein substitutes HCU Lophlex Powder, MSUD Lophlex Powder and TYR Lophlex Powder, which are the powdered versions of the currently existing and already ACBS approved HCU Lophlex LQ, MSUD Lophlex LQ and TYR Lophlex LQ. The powder format allows patients to make up their protein substitutes at different volumes/flavour strengths to aid acceptability and/or tolerance. These Lophlex Powders are composed of adapted mixtures of other essential and non-essential amino acids (excluding homocysteine in HCU Lophlex Powder, branched chain amino acids in MSUD Lophlex Powder and tyrosine in TYR Lophlex Powder), carbohydrates, fats, vitamins and minerals, and are enriched with Docosahexaenoic acid (DHA). The amino acids in the HCU, MSUD and TYR Lophlex Powders provide 20g protein equivalent (PE) per 29g (for HCU) and 28g (for MSUD and TYR) sachets.\n\nThe case studies aim to evaluate the acceptability, compliance and gastro-intestinal tolerance of these powdered protein substitutes. Patients will receive the condition specific case study product for 28 days and will have 2 conversations with the Dietitian during this period. For this project we aim to recruit 10 patients per condition.
REC name
South West - Frenchay Research Ethics Committee
REC reference
19/SW/0091
Date of REC Opinion
26 Jun 2019
REC opinion
Further Information Favourable Opinion