Evaluation of PKU Sphere Liquid

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)

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.

Study Overview

• Status: Completed
• Conditions: Phenylketonurias
• Intervention / Treatment: Dietary supplement: PKU sphere liquid

Detailed 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.

• Study Type: Interventional
• Enrollment (Actual): 19
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United Kingdom
  • Belfast, United Kingdom, BT12 6BA
    • Royal Victoria Hospital
  • Birmingham, United Kingdom, B4 6NH
    • Birmingham Children's Hospital
  • Sheffield, United Kingdom, S5 7AU
    • Northern General Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: PKU sphere liquid; PKU sphere liquid will be prescribed by the study dietitian based on the patient's individual requirements.	Dietary supplement: PKU sphere liquid; PKU sphere liquid will be prescribed by the study dietitian based on the patient's individual requirements.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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
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

Collaborators and Investigators

• Sponsor: Vitaflo International, Ltd
• Investigators: Principal Investigator: Anita MacDonald, Birmingham Women's and Children's NHS Foundation Trust

Study record dates

Study Major Dates

• Study Start (Actual): November 23, 2021
• Primary Completion (Actual): June 21, 2022
• Study Completion (Actual): February 28, 2023

Study Registration Dates

• First Submitted: October 14, 2021
• First Submitted That Met QC Criteria: October 14, 2021
• First Posted (Actual): October 27, 2021

Study Record Updates

• Last Update Posted (Actual): February 16, 2024
• Last Update Submitted That Met QC Criteria: February 15, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Keywords: PKU; Sphere; Liquid
• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Amino Acid Metabolism, Inborn Errors; Phenylketonurias
• Other Study ID Numbers: MCT-GMPL-2018-02-21; 271801 (Other Identifier: IRAS); 20/NW/0273 (Other Identifier: HRA)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No