Evaluation of PKU Sphere Liquid

Phenylketonurias Clinical Trial

Official title:

A Study to Evaluate the Acceptability, Tolerance and Adherence of Children and Adults Consuming PKU Sphere Liquid, a Food for Special Medical Purposes (FSMP), for the Dietary Management of Phenylketonuria (PKU)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05096988
• Other study ID #: MCT-GMPL-2018-02-21
• Secondary ID: 27180120/NW/0273
• Status: Completed
• Phase: N/A
• Start date: November 23, 2021
• Est. completion date: February 28, 2023

Study information

• Verified date: February 2024
• Source: Vitaflo International, Ltd
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

PKU Sphere Liquid is a prospective, open-label, acceptability study to evaluate PKU sphere liquid in up to 15 participants aged 3 and above for the dietary management of PKU over 31 days.

Description:

PKU Sphere Liquid is a newly-developed Food for Special Medical Purposes (FSMP) designed for the dietary management of patients with phenylketonuria (PKU).

PKU sphere liquid is a vanilla flavoured, ready-to-drink, low phenylalanine protein substitute containing a blend of casein glycomacropeptide (GMP) isolate, essential and non-essential amino acids, carbohydrate, fat, vitamins, minerals, and Docosachexaenoic acid (DHA).

This is a feasibility study designed to evaluate the acceptability, tolerance and adherence of children and adults consuming PKU sphere liquid.

Participants will be given a four-week supply of PKU Sphere Liquid and they will be asked to complete a daily diary and short questionnaire for four weeks to record information on: adherence, gastrointestinal tolerance, palatability and how the product is used.

Each participant will be on the trial for 31 days. This includes a 3-day baseline period and 28 days taking the study product. If deemed appropriate in each individual circumstance, the sponsor of the trial, Vitaflo International Ltd, will continue to supply the product free of charge at the end of the trial until available on prescription.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 19
• Est. completion date: February 28, 2023
• Est. primary completion date: June 21, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years and older

Eligibility

Inclusion Criteria:

• Diagnosis of PKU requiring a low protein diet and protein substitute.

• Aged three (3) years and over

• Already taking a minimum of one GMP based protein substitute per day or have previously taken one

• Able to comply with the study protocol and take the study product, according to the opinion of the PI.

• Willingly given, written, informed consent from patient or parent/guardian.

• Willingly given, written assent (if appropriate).

Exclusion Criteria:

• Inability to comply with the study protocol, in the opinion of the investigator.

• Any co-morbidity, which, in the opinion of the investigator, would preclude participation in the study.

• Diagnosis of persistent hyperphenylalaninaemia, or mild PKU not requiring dietary intervention with a low protein diet and protein supplement.

• Unwilling/unable to take a GMP based protein substitute.

• Women who are pregnant / breastfeeding at the start of the study or planning to become pregnant during the study period.

N.B.: Women who become pregnant unexpectedly during this study will be immediately withdrawn from the study.

Related Conditions & MeSH terms

• Phenylketonurias

Intervention

Dietary Supplement:
• PKU sphere liquid
PKU sphere liquid will be prescribed by the study dietitian based on the patient's individual requirements.

Locations

Country	Name	City	State
United Kingdom	Royal Victoria Hospital	Belfast
United Kingdom	Birmingham Children's Hospital	Birmingham
United Kingdom	Northern General Hospital	Sheffield

Sponsors (1)

Lead Sponsor	Collaborator
Vitaflo International, Ltd

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Questionnaire of self-reported adherence to the prescribed amount of study product	Assessment of participant's adherence to prescribed amount during the eight week intake of the study product	4 weeks
Primary	Product acceptability rated on a Likert scale by the participant after four week intake	Assessment of participant's acceptability following four week intake of the study product Scale is from 1 to 5: 1 = Loved it, 5 = Really didn't like it	4 weeks
Primary	Questionnaire of self-reported changes in gastrointestinal tolerance during four week intake	Assessment of participant's gastrointestinal tolerance during the four week intake of the study product	4 weeks