GMP Drink for PKU Study

Nutricia UK Ltd40 enrolled

Overview

This study will evaluate the safety, gastrointestinal (GI) tolerance, acceptability and compliance of a Glycomacropeptide-based protein substitute for patients with Phenylketonuria (PKU).

Study Type

INTERVENTIONAL

Allocation

Purpose

SUPPORTIVE_CARE

Masking

NONE

Enrollment

Conditions

Phenylketonuria

Interventions

Glycomacropeptide-based protein substituteDIETARY_SUPPLEMENT

Eligibility

Sex: ALLMin age: 3 Years

Medical Language ↔ Plain English

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

Locations (5)

Great Ormond Street Hospital

London, United Kingdom

Guy's and St Thomas' NHS Fondation Trust

London, United Kingdom

UCLH

London, United Kingdom

Royal Manchester Children's Hospital

Manchester, United Kingdom

Outcomes

Primary Outcomes

Blood Phenylalanine (and other amino acids)

Blood spot test

Time frame: 3 points throughout 28 days

Secondary Outcomes

Gastro-intestinal tolerance questionaire

Questionaire detailing any GI symptoms, severity and change from usual

Time frame: 12/31 days

Product compliance questionaire

Breif questionaire on amounts offered and amounts actually consumed, compared to recomended amount.

Time frame: 31 days

Product acceptability questionnaire

Breif tick-box questionaire on overal liking and acceptability of product

Time frame: 2/31 days

Patient reproted nutrient intake (3 day food diaries)

3 day food diaries, subsequently analysed in dietary software.

Time frame: 6/31 days

Pateint-reported appetite (visual analogue scale)

Measures of overal appetite and satiety via visual analouge scale

Time frame: 6/31 days

Antropometry (hieight and weight)

Measurements of height and weight at baseline and end of study

Time frame: 2/31 days

Safety (Adverse events reporting)

Reporting of adverse events throughout study

Time frame: 31 days

Data from ClinicalTrials.gov

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