Menu
Recruiting NCT06941532

GMP Powdered Substitutes in PKU and TYR

No phase Interventional Phenylketonuria Tyrosinemia

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: GMP Intervention Product.
Who it may be relevant to
Registry conditions: Phenylketonuria, Tyrosinemia. Basic parameters: from 3 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United Kingdom
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

Evaluating the Adherence, Tolerance, Acceptability and Safety of New GMP-based Powdered Protein Substitutes in PKU and TYR: a Case Study Series

Overview

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.

Interventions

  • Dietary supplement GMP Intervention Product
    Condition specific GMP based intervention product

Primary outcome measures

  • Gastro-intetsinal tolerance [Time frame: Measured at baseline (day 1) and end of intervention (day 29)]
Secondary outcome measures (6)
  • Acceptability [Time frame: Measured at baseline (day 1) and end of intervention (day 29)]
  • Compliance [Time frame: Measured at baseline (day 1) and end of intervention (day 29)]
  • Blood phenylalanine, blood tyrosine and other amino acid levels [Time frame: Day 1 (baseline) and day 29 (end of intervention)]
  • Anthropometry [Time frame: Day 1 (baseline) and day 29 (end of intervention)]
  • Patient history [Time frame: Day 1 (baseline) and day 29 (end of intervention)]
  • Safety/ Adverse Events [Time frame: Throughout the case study series (day 1 to day 29).]

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Basic science

Study locations

United Kingdom · 1 center
  • Birmingham Women and Children's Hospital — Birmingham

Identifiers

NCT: NCT06941532 · GMP2024

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗