GMP drink for PKU study


Phenylketonuria (PKU)

Full Study Name

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

Study ID



Nutricia UK Ltd

Lead Site

Great Ormond Street Hospital

Chief Investigator

Rebecca Stratton

Local Principal Investigator

Karen Van Wyk

Study Status


Main Aims

To assess if a GMP based nutritional supplement (plus modified diet) can be used in place of a traditional amino acid based supplement (plus modified diet) to help support adequate blood phenylalanine control for patients over 3 years of age with PKU

Inclusion Criteria
  • Male or female
  • Over 3 years of age
  • Diagnosed with classical or variant type phenylketonuria
  • Have been compliant in taking at least one amino acid-based, Phe-free protein substitute, providing at least 20g protein equivalents, for at least 1 month prior to trial commencement
  • Have a prescribed daily Phe allowance
  • Written informed consent from patient, or from parent / carer if applicable
Exclusion Criteria
  • Currently prescribed Sapropterin or similar tetrahydrobiopterin based medication
  • Pregnant or lactating
  • Requiring parenteral nutrition
  • Major hepatic or renal dysfunction
  • Participation in other studies within 1 month prior to entry of this study
  • Allergy to any of the study product ingredients, including milk protein or soya
  • Investigator concern around willingness/ability of patient or parent/carer to comply with protocol requirements
Open Sites

Manchester, Great Ormond Street, Guy’s and St Thomas’ NHS Fondation Trust, University College London Hospital, Sheffield Teaching Hospitals

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