PKU Low Calorie Drink Study

Nutricia UK Ltd

Overview

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).

Study Type

INTERVENTIONAL

Allocation

Purpose

SUPPORTIVE_CARE

Masking

NONE

Conditions

Phenylketonurias Hyperphenylalaninaemia

Interventions

Low calorie protein substituteDIETARY_SUPPLEMENT

The evaluated dietary supplement is a lower calorie amino acid based liquid protein substitute designed for patients with PKU.

Eligibility

Sex: ALLMin age: 3 Years

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

Compliance

Questionnaire on amounts offered and amounts actually consumed, compared to recommended amount. Amounts (values) are recorded

Time frame: Daily for 31 days

Secondary Outcomes

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)

Time frame: Recorded at baseline and end of intervetnion (day 31)

Blood phenylalanine and other amino acid levels

Blood spot test.

Time frame: 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.

Time frame: recorded at baseline, beginning of intervention and end of intervention(12 out of 31 days in total)

Nutrient intake

24h dietary recall.

Time frame: Recorded at basleine and end of intervention (day 31)

Anthropometry

Measurements of height (m) and weight (kg) at baseline and end of study.

Time frame: Recorded at baseline and end of intervention (day 31)

Data from ClinicalTrials.gov

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