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RECRUITINGINTERVENTIONAL

GMP Powdered Substitutes in PKU and TYR

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

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

Who May Qualify: - 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 willing to sign a consent form 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 Who Should NOT Join This Trial: - 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 Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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

Treatments Being Tested

DIETARY_SUPPLEMENT

GMP Intervention Product

Condition specific GMP based intervention product

Locations (1)

Birmingham Women and Children's Hospital
Birmingham, United Kingdom