GMP Powdered Substitutes in PKU and TYR

Nutricia

Status

Not yet enrolling

Conditions

Tyrosinemia

Phenylketonuria

Treatments

Dietary Supplement: GMP Intervention Product

Study type

Interventional

Funder types

Industry

Identifiers

NCT06941532

GMP2024

Details and patient eligibility

About

Four new GMP-based protein substitutes have been developed to support the dietary management of PKU and TYR. These products are powdered protein substitutes, low in phenylalanine and low in phenylalanine and tyrosine respectively, with a mix of glycomacropeptide (GMP), essential and non-essential amino acids, carbohydrates, fibres, fats (including DHA) and micronutrients. The proteins in these new protein substitutes are based on GMP, a peptide isolated from milk during cheese manufacturing and the only known naturally derived protein source low in phenylalanine and tyrosine. The GMP is supplemented with other amino acids which are naturally low or not present in GMP, as well as carbohydrates, fats, fibre and micronutrients. Studies to date have illustrated that PKU patients who replace their regular phenylalanine-free amino acid-based formula with GMP-based foods may have better diet compliance and prefer the taste whilst maintaining metabolic control.

This series of case-studies aims to evaluate the gastrointestinal tolerance, acceptability, compliance, and safety of these four GMP based protein substitutes in both adults and children over 3 years. These case studies will last 29 days in total, including a 1-day baseline period followed by a 28-day intervention period. The case studies will be conducted across multiple specialist metabolic centres in the UK. A series of case studies is undertaken due to the rarity of these conditions, the diversity of patient types and the difficulty in recruiting these patients to trials.

Enrollment

40 estimated patients

Sex

All

Ages

3+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Male or female
Over 3 years of age
Patients diagnosed with any phenotype of PKU characterised by hyperphenylalaninaemia or patients diagnosed with tyrosinaemia
Have been compliant in taking at least one protein substitute, providing at least 15g protein equivalents, for at least 1 month prior to trial commencement
Have a prescribed daily phenylalanine or phenylalanine and tyrosine allowance for PKU or TYR respectively
Written or electronic informed consent from patient, and/or from parent/caregiver if applicable
Participants who are anticipated to be able to take at least one sachet of GMP Product per day

Exclusion criteria

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
Investigator concern around willingness/ability of patient or parent/caregiver to comply with protocol requirement