PKU Low Calorie Drink Study

Not Applicable

Withdrawn

• Conditions: Hyperphenylalaninaemia; Phenylketonurias
• Interventions: Dietary Supplement: Low calorie protein substitute

• Registration Number: NCT04272736
• Lead Sponsor: Nutricia UK Ltd

Brief Summary

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).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-08-29
• Study First Submitted QC: 2020-02-14
• Study First Posted: 2020-02-17
• Study Start: 2023-12-01
• Primary Completion: 2023-12-31
• Study Completion: 2023-12-31
• Status Verified: 2025-02
• Last Update Submitted: 2025-02-24
• Last Update Posted: 2025-02-26

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: 30

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Low calorie protein substitute	Low calorie protein substitute	Single arm designed, 3 day baseline, 28 day on the lower calorie amino acid based liquid protein substitute.

Primary Outcome Measures

Name	Time	Method
Compliance	Daily for 31 days	Questionnaire on amounts offered and amounts actually consumed, compared to recommended amount. Amounts (values) are recorded

Secondary Outcome Measures

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