GMP drink for PKU study
Research type
Research Study
Full title
Evaluating the tolerance, safety and acceptability of PKU GMPro, a whey protein derived feed for the dietary management of phenylketonuria in children and adults – A pilot trial
IRAS ID
198734
Contact name
Robert Browne
Contact email
Sponsor organisation
Nutricia Ltd UK
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 4 months, 31 days
Research summary
The dietary management of Phenylketonuria (PKU) involves restricting dietary Phenylalanine (Phe) intake to promote optimal blood Phe control, whilst meeting all other nutrient requirements. As Phe is present in nearly all dietary proteins, this typically requires a very low protein diet, supplemented with a prescribed Phe-free amino acid (AA) formula. However compliance to this diet is often poor, with patients commonly going “off diet” during adolescence. This can lead to entirely preventable, lifelong mental impairments.
PKU GMPro is a vanilla flavour protein substitute drink (either in powdered or ready-to-drink format) for the dietary management of PKU in children over 3 years and adults. The protein in PKU GMPro is based on Glycomacropeptide (GMP), a peptide isolated from whey during cheese manufacturing and the only known naturally derived protein source which is very low in Phe. The GMP is then supplemented with other AAs which are low or not present in GMP, alongside fats, carbohydrates, vitamins and minerals to produce a more nutritionally balanced dietary supplement. This differs from traditional PKU formulae which are reliant mainly on individual, synthetically produced amino acids. Studies to date have illustrated that patients replacing their AA formula with GMP based foods have better diet compliance, prefer the taste and may have improved blood Phe control.
This study will evaluate the safety, gastrointestinal tolerance, acceptability and compliance of PKU GMPro in 80 children (aged 3+) and adults with classical or variant PKU, over a 31 day period. The primary outcome will be blood Phe level (alongside other amino acids), with secondary outcomes of compliance, acceptability, nutrient intake, satiety, spread of prescription throughout the day and anthropometry. The study is conducted for tolerance and safety purposes to meet the UK ACBS requirements for acceptability studies. The feed is a Food for Special Medical Purposes and MHRA exempt.
REC name
London - Central Research Ethics Committee
REC reference
16/LO/0543
Date of REC Opinion
1 Apr 2016
REC opinion
Favourable Opinion