PKU Low Calorie Drink Study

Phenylketonurias Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT04272736
• Other study ID #: PKULite2018
• Status: Not yet recruiting
• Phase: N/A
• Start date: December 1, 2023
• Est. completion date: December 31, 2023

Study information

• Verified date: April 2023
• Source: Nutricia UK Ltd
• Contact: Gary Hubbard, PhD
• Phone: 07738024720
• Email: gary.hubbard[@]nutricia.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 30
• Est. completion date: December 31, 2023
• Est. primary completion date: December 31, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Hyperphenylalaninaemia
• Phenylketonurias

Intervention

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

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Nutricia UK Ltd

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Compliance	Questionnaire on amounts offered and amounts actually consumed, compared to recommended amount. Amounts (values) are recorded	Daily for 31 days
Secondary	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)
Secondary	Blood phenylalanine and other amino acid levels	Blood spot test.	Recorded at baselien and end of intervention (day 31)
Secondary	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)
Secondary	Nutrient intake	24h dietary recall.	Recorded at basleine and end of intervention (day 31)
Secondary	Anthropometry	Measurements of height (m) and weight (kg) at baseline and end of study.	Recorded at baseline and end of intervention (day 31)