GMP Drink for PKU Study

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

Official Title
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Evaluating the Tolerance, Safety and Acceptability of PKU GMPro, a Whey Protein Derived Feed for the Dietary Management of Phenylketonuria in Children and Adults - a Pilot Trial

Conditions
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Phenylketonuria

Intervention / Treatment
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* Dietary Supplement: Glycomacropeptide-based protein substitute


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 3 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Supportive Care
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: GMP<br>Single arm designed, 3 day baseline, 28day on GMP | Dietary Supplement: Glycomacropeptide-based protein substitute<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Blood Phenylalanine (and other amino acids) | Blood spot test | 3 points throughout 28 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Gastro-intestinal tolerance questionaire | Questionaire detailing any GI symptoms, severity and change from usual | 12/31 days | 
| Product compliance questionaire | Breif questionaire on amounts offered and amounts actually consumed, compared to recomended amount. | 31 days | 
| Product acceptability questionnaire | Breif tick-box questionaire on overal liking and acceptability of product | 2/31 days | 
| Patient reproted nutrient intake (3 day food diaries) | 3 day food diaries, subsequently analysed in dietary software. | 6/31 days | 
| Pateint-reported appetite (visual analogue scale) | Measures of overal appetite and satiety via visual analouge scale | 6/31 days | 
| Antropometry (hieight and weight) | Measurements of height and weight at baseline and end of study | 2/31 days | 
| Safety (Adverse events reporting) | Reporting of adverse events throughout study | 31 days |