PKU Synergy Evaluation
Research type
Research Study
Full title
Evaluating the efficacy of PKU Synergy in patients expressing phenylketonuria or hyperphenylalaninemia
IRAS ID
223150
Contact name
Benjamin Green
Contact email
Sponsor organisation
Nutricia Ltd.
Duration of Study in the UK
2 years, 4 months, 31 days
Research summary
In clinical practice, the objective of phenylketonuria management is to prevent profound and irreversible intellectual disability while ensuring growth and physical development is not compromised through the avoidance of nutritional deficiencies. Management of phenylketonuria therefore centres on a strict low-protein diet virtually free of phenylalanine, where free/low phenylalanine protein substitutes are integral aides. A low-phenylalanine diet, however, can be burdensome, demanding and difficult to follow. This is especially true in adults. Consequently, many patients tend to relax and/or stop adhering to prescribed dietary and protein substitute regimens. Micronutrient inadequacies are therefore common and in some instances, inevitable.
Following dietary and/or protein substitute relaxation many patients find it difficult to return to full compliance, which may have included 3-4 protein substitutes daily. In such patients, a one-a-day phenylalanine-free protein substitute could prove a promising management strategy to improve compliance. Indeed, it would be particularly advantageous if the phenylalanine-free protein substitute delivers an increased concentration of nutrients that are particularly low in these patients compared to currently available PKU products. PKU Synergy is a new one-a-day phenylalanine-free protein substitute designed for the dietary management of proven phenylketonuria or hyperphenylalaninemia in poorly compliant adults with an increased phenylalanine-tolerance/intake. As such, this study aims to evaluate the efficacy (changes relating to nutritional status and metabolic control) of PKU Synergy, while determining the tolerance, compliance, safety and acceptability in phenylketonuria patients. The study will be conducted over 31 days. In addition to appropriate nutritional management, fifty adults (≥ 16 years) will be recruited and randomly allocated to one of the two intervention arms (n = 25 per arm) and receive either one sachet of PKU Synergy daily or continue their usual dietary and/or protein substitute regimen (maximum of 1 protein substitute per day (equal to 20g protein equivalent)) for 28 days.
REC name
East of England - Cambridge East Research Ethics Committee
REC reference
17/EE/0078
Date of REC Opinion
6 Mar 2017
REC opinion
Further Information Favourable Opinion