Effects of an innovative GMP-based protein supplement in pediatric PKU
Research type
Research Study
Full title
The effects of an innovative Glycomacropeptide based protein supplement purified to lower the phenylalanine content for the treatment of paediatric patients with Phenylketonuria.
IRAS ID
279630
Contact name
Anita MacDonald
Contact email
Sponsor organisation
Cambrooke UK Ltd
Duration of Study in the UK
1 years, 0 months, 1 days
Research summary
This is a two-stage, 15-week randomized crossover trial in 18 phenylketonuria (PKU) children. The objective of the study is to evaluate the efficacy of a low Phe diet combined with a Glycomacropeptide (GMP)-based protein substitute (PS) in the treatment of paediatric patients with PKU. GMP, a very low-Phe peptide, is used to develop protein substitutes low in Phe with the right mix of amino acids (AA), vitamins and minerals. Currently the recommendation in the UK for the PKU paediatric patients is to use GMP-based PS only partly (up to 70% of total daily PS intake) in combination with AA-based PS. In the proposed study, the efficacy of a low-Phe diet combined with a purified GMP-based PS used at 100% will be evaluated in children with PKU aged 5 to 16 years. The participants will visit the clinic at the start and end of each arm of the study for the dietitian to obtain anthropometric data and for the participant to complete questionnaires regarding mood, attention, hunger, satiety, GI symptoms, and acceptance of GMP-based PS. Blood Phe will be measured biweekly during the study period by means of a dried blood spot. Following a 4 week baseline period on their usual PS, the subjects will be randomly allocated to a GMP or an AA group for 4 weeks. At the end of this period, both groups will have a 2-week washout period consuming their usual PS. At the end of the washout period the GMP-based group will become the AA-based group and vice versa for 4 weeks. 3-day food diaries will be completed at baseline, end of phase 1, pre-phase 2 and end of phase 2. The change in blood Phe in patients ingesting the GMP-based PS compared to the AA-based PS will be determined as the difference at the end of each study arm and the baseline Phe prior to the beginning of the study. Upon completion of the study, the patients can choose to remain on the GMP-based PS, return to their usual PS or utilize a combination of both.
REC name
West Midlands - South Birmingham Research Ethics Committee
REC reference
22/WM/0019
Date of REC Opinion
11 Mar 2022
REC opinion
Further Information Favourable Opinion