PKU Lophlex Evaluation
Research type
Research Study
Full title
Compliance, tolerance and acceptability of a tablet protein substitute for the dietary management of phenylketonuria
IRAS ID
233489
Contact name
Rebecca Stratton
Contact email
Sponsor organisation
Nutricia Ltd.
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
CPMS ID: 36117, NIHR Portfolio
Duration of Study in the UK
2 years, 2 months, 4 days
Research summary
Management of phenylketonuria (PKU) centres on a strict low-protein diet virtually free of phenylalanine, where free/low phenylalanine protein substitutes are integral aides. Protein substitutes are typically presented in powder format and reconstituted in water to a set volume, but may also be offered in ready-to-drink or tablet formats. Despite the need to consume large quantities, protein substitutes in tablet format may help overcome numerous organoleptic issues compared to powdered or ready-to-drink preparations, but may also offer additional benefits including convenience and reduced preparation.
At present, tablet-based protein substitutes only offer a balanced mixture of essential and non-essential amino acids. Considering the daily burden and discipline required to follow the restrictive low-protein diet, reports suggest achieving full compliance with tablet-based protein substitutes is difficult, and this becomes especially challenging when given alongside prescribed multivitamin supplements. In this sense, patients consuming tablet protein substitutes may be required to consume 70-140 tablets per day (average = 75 tablets daily) alongside an additional 5 prescribable vitamin and mineral tablets. Developed specifically for the dietary management of proven PKU, PKU Lophlex tablets comprise an adapted mixture of essential and non-essential amino acids (excluding phenylalanine) and are combined with vitamins, minerals and trace elements therefore offering compositional advantages and hopefully reducing patient burden.
This intervention trial aims to evaluate the acceptability (compliance, gastro-intestinal tolerance and palatability) of PKU Lophlex tablets, while also capturing information related to blood amino acid profiles, dietary intake, anthropometry and safety as part of a low phenylalanine dietary regimen in PKU patients. Thirty eligible PKU patients (≥ 8 years) will be recruited. Patients will firstly observe a 7-day baseline period. During baseline, patients will continue with their existing diet and protein substitute prescription before receiving PKU Lophlex Tablets daily for 28 days in addition to appropriate nutritional management.
REC name
West Midlands - South Birmingham Research Ethics Committee
REC reference
18/WM/0082
Date of REC Opinion
13 Apr 2018
REC opinion
Further Information Favourable Opinion