Evaluation of PKU Explore (PKU Explore)

A Study to Evaluate the Acceptability of PKU Explore, a Renovated Phenylalanine Free Second Stage Protein Substitute for Use in the Dietary Management of Phenylketonuria in Infants From 6 Months to 5 Years of Age With Regard to Product Tolerance and Adherence.

To evaluate the acceptability, tolerance and effect on metabolic control of PKU Explore, a renovated Phe free protein substitute for the dietary management of PKU in children from 6 months to 5 years.

Study Overview

• Status: Completed
• Conditions: Phenylketonuria; Inborn Errors of Metabolism
• Intervention / Treatment: Dietary supplement: PKU Explore

Detailed Description

This is a prospective, acceptability study to evaluate the gastrointestinal tolerance, palatability and adherence of PKU explore in a four-week period for 15 participants aged between 6 months and 5 years for the dietary management of PKU.

The outcome of this assessment will be used in a submission to the regulatory authorities, Advisory Committee on Borderline Substances (ACBS), for PKU Start to become reimbursable on prescription in the UK.

The participant's dietitian will advise on an appropriate amount of PKU Explore based on individual requirements. Parents/carers will be asked to substitute their usual Phe-free protein substitute with PKU Explore for one (1) month. The sponsor will supply PKU Explore free of charge.

Parents/guardians will be asked to record information about the following:

Daily PKU Explore intake Stools Vomiting and Spit-up Feed / Fluid Intake Compliance Phenylalanine Levels (from their routine blood spot testing)

Final Evaluation about the presentation of the product, ease of preparation and use.

• Study Type: Interventional
• Enrollment (Actual): 22
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United Kingdom
  • Lanarkshire
    • Glasgow, Lanarkshire, United Kingdom, G51 4TF
      • NHS Greater Glasgow and Clyde
  • West Midlands
    • Burringham, West Midlands, United Kingdom, B4 6NH
      • Birmingham Women's and Children's NHS Foundation Trust
  • West Yorkshire
    • Bradford, West Yorkshire, United Kingdom, BD5 0NA
      • Bradford Teaching Hospitals NHS Foundation Trust

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 months to 5 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

i. A diagnosis of PKU on new-born screening requiring a low protein diet and Phe-free L-amino acid supplements.

ii. Aged between 6 months and 5 years.

iii. Already taking a second stage concentrated protein substitute as part of their PKU management OR is at the stage in their PKU management when a second stage concentrated protein substitute is recommended to commence.

iv. Willingly given, written, informed consent from parent/guardian.

v. Willingly given, written assent (if appropriate).

Exclusion Criteria:

i. Diagnosis of persistent hyperphenylalaninaemia, or mild PKU not requiring dietary treatment with a low protein diet and Phe-free L-amino acid supplements.

ii. Diagnosis of a concurrent condition which may adversely affect developmental progression and feeding ability.

iii. Participation in any other clinical trial/acceptability study.

iv. Any serious medical precluding the study intervention.

v. Use of additional macro/micronutrient supplements during the study period, unless clinically indicated and prescribed by the investigator (must be recorded in patient case record file).

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: Consumption of PKU Explore; Daily feed, substituting the participant's normal phe-free protein substitute for PKU Explore.	Dietary supplement: PKU Explore; PKU Explore is an unflavoured, powdered, phenylalanine-free, protein substitute, containing essential and non-essential amino acids, carbohydrate, sugar, vitamins, minerals, trace elements and the long chain polyunsaturated fatty acids (LCPs); arachidonic acid (AA) and docosahexaenoic acid (DHA).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Product compliance daily diary	Quantitative assessments from subject questionnaires that allow evaluation of compliance with the study product, i.e. actual versus prescribed intake.	Days 1-28
GI tolerance daily diary assessing change	Qualitative assessments from subject questionnaires that allow evaluation of any change in the gastro-intestinal tolerance of the study product.	Days 1-7 and 22-28
Ease of use questionnaire	Qualitative assessment from subject questionnaire that allows evaluation of the ease of use of the study product.	Day 29
Weekly phenylalanine control	Collection of quantitative data regarding phenylalanine control using routine biochemical testing	Days 1-28

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Actual): June 8, 2017
• Primary Completion (Actual): March 15, 2018
• Study Completion (Actual): March 15, 2018

Study Registration Dates

• First Submitted: May 24, 2017
• First Submitted That Met QC Criteria: May 25, 2017
• First Posted (Actual): May 30, 2017

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: Acceptability; Metabolic; Control; Metabolism; Phenylketonuria; PKU
• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Amino Acid Metabolism, Inborn Errors; Phenylketonurias; Metabolism, Inborn Errors
• Other Study ID Numbers: MCT-PKUE-2016-12-05; 219119 (Other Identifier: IRAS)

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