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Evaluation of PKU Sphere Liquid

Primary Purpose

Phenylketonurias

Status
Completed
Phase
Not Applicable
Locations
United Kingdom
Study Type
Interventional
Intervention
PKU sphere liquid
Sponsored by
Vitaflo International, Ltd
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Phenylketonurias focused on measuring PKU, Sphere, Liquid

Eligibility Criteria

3 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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N.B.: Women who become pregnant unexpectedly during this study will be immediately withdrawn from the study.

Sites / Locations

  • Royal Victoria Hospital
  • Birmingham Children's Hospital
  • Northern General Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

PKU sphere liquid

Arm Description

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

Outcomes

Primary Outcome Measures

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
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
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

Secondary Outcome Measures

Full Information

First Posted
October 14, 2021
Last Updated
August 18, 2023
Sponsor
Vitaflo International, Ltd
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1. Study Identification

Unique Protocol Identification Number
NCT05096988
Brief Title
Evaluation of PKU Sphere Liquid
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)
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Completed
Study Start Date
November 23, 2021 (Actual)
Primary Completion Date
June 21, 2022 (Actual)
Study Completion Date
February 28, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vitaflo International, Ltd

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Phenylketonurias
Keywords
PKU, Sphere, Liquid

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
19 (Actual)

8. Arms, Groups, and Interventions

Arm Title
PKU sphere liquid
Arm Type
Experimental
Arm Description
PKU sphere liquid will be prescribed by the study dietitian based on the patient's individual requirements.
Intervention Type
Dietary Supplement
Intervention Name(s)
PKU sphere liquid
Intervention Description
PKU sphere liquid will be prescribed by the study dietitian based on the patient's individual requirements.
Primary Outcome Measure Information:
Title
Questionnaire of self-reported adherence to the prescribed amount of study product
Description
Assessment of participant's adherence to prescribed amount during the eight week intake of the study product
Time Frame
4 weeks
Title
Product acceptability rated on a Likert scale by the participant after four week intake
Description
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
Time Frame
4 weeks
Title
Questionnaire of self-reported changes in gastrointestinal tolerance during four week intake
Description
Assessment of participant's gastrointestinal tolerance during the four week intake of the study product
Time Frame
4 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Anita Macdonald
Organizational Affiliation
Birmingham Women's and Children's NHS Foundation Trust
Official's Role
Principal Investigator
Facility Information:
Facility Name
Royal Victoria Hospital
City
Belfast
ZIP/Postal Code
BT12 6BA
Country
United Kingdom
Facility Name
Birmingham Children's Hospital
City
Birmingham
ZIP/Postal Code
B4 6NH
Country
United Kingdom
Facility Name
Northern General Hospital
City
Sheffield
ZIP/Postal Code
S5 7AU
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No

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Evaluation of PKU Sphere Liquid

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