GMP Powdered Substitutes in PKU and TYR

NCT ID: NCT06941532

Last Updated: 2025-12-19

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: NA
• Total Enrollment: 40 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-05-20
• Study Completion Date: 2026-12-31

Brief Summary

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.

Conditions

Phenylketonuria; Tyrosinemia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: BASIC_SCIENCE
• Blinding Strategy: NONE

Study Groups

GMP intervention product

Group Type: EXPERIMENTAL

GMP Intervention Product

Intervention Type: DIETARY_SUPPLEMENT

Condition specific GMP based intervention product

Interventions

GMP Intervention Product

Condition specific GMP based intervention product

Intervention Type: DIETARY_SUPPLEMENT

Eligibility Criteria

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

• Minimum Eligible Age: 3 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Nutricia UK Ltd

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Gary Hubbard, Dr

Role: STUDY_DIRECTOR

Nutricia UK Ltd

Locations

Birmingham Women and Children's Hospital

Birmingham, , United Kingdom

Site Status: RECRUITING

Countries

United Kingdom

Central Contacts

Ellen Taylor

Role: CONTACT

Phone: 07900724091

Email: ellen.taylor@nutricia.com

Other Identifiers

GMP2024

Identifier Type: -

Identifier Source: org_study_id