PKU Low Calorie Drink Study

Study Overview
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Brief Summary
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This study will evaluate the compliance, acceptability, gastrointestinal (GI) tolerance and safety of a lower calorie amino acid based liquid protein substitute for patients with Phenylketonuria (PKU) or hyperphenylalaninemia (HPA).

Official Title
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Evaluating the Compliance, Acceptability, Safety and Tolerance of a Lower Calorie Protein Substitute for the Dietary Management of Phenylketonuria in Children and Adults - a Pilot Study

Conditions
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Phenylketonurias, Hyperphenylalaninaemia

Intervention / Treatment
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* Dietary Supplement: Low calorie protein substitute


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male or female 3 years of age or above Diagnosed with classical or variant type phenylketonuria (PKU), or hyperphenylalaninemia (HPA) Have been compliant in taking at least one amino acid-based, Phe-free protein substitute, providing at least 20g protein equivalent daily, for at least 1 month prior to study commencement Have a prescribed daily Phe allowance Written informed consent from patient, or from parent / carer if applicable Exclusion Criteria: Pregnant or lactating Requiring enteral tube or parenteral nutrition Major hepatic or renal dysfunction Participation in other studies within 1 month prior to entry to this study Allergy to any of the study product ingredients Investigator concern around the patient being underweight (e.g.: BMI lower than 18.5kg/m2 for adults) and/or having an eating disorder 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: Low calorie protein substitute<br>Single arm designed, 3 day baseline, 28 day on the lower calorie amino acid based liquid protein substitute. | Dietary Supplement: Low calorie protein substitute<br>* The evaluated dietary supplement is a lower calorie amino acid based liquid protein substitute designed for patients with PKU.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Compliance | Questionnaire on amounts offered and amounts actually consumed, compared to recommended amount. Amounts (values) are recorded | Daily for 31 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Acceptability: Tick-box questionnaire | Tick-box questionnaire on overall liking and acceptability of product (ie dislike a lot, dislike moderately, neutral, like moderately, like a lot) | Recorded at baseline and end of intervetnion (day 31) | 
| Blood phenylalanine and other amino acid levels | Blood spot test. | Recorded at baselien and end of intervention (day 31) | 
| Gastro-intestinal tolerance | Questionnaire detailing any GI symptoms, severity (none, mild, moderate, severe) and change from usual. | recorded at baseline, beginning of intervention and end of intervention(12 out of 31 days in total) | 
| Nutrient intake | 24h dietary recall. | Recorded at basleine and end of intervention (day 31) | 
| Anthropometry | Measurements of height (m) and weight (kg) at baseline and end of study. | Recorded at baseline and end of intervention (day 31) |