GMP drink for PKU study

Condition

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

GMP2016

Sponsor

Nutricia UK Ltd

Lead Site

Great Ormond Street Hospital

Chief Investigator

Rebecca Stratton

Local Principal Investigator

Karen Van Wyk

Study Status

recruiting

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

For more information click here