Evaluation of PKU Explore France

An Exploratory Study to Evaluate the Acceptability of PKU Explore, a Food for Special Medical Purposes, for Use in the Dietary Management of Phenylketonuria in Infants From 6 Months to 3 Years of Age With Regard to Product-acceptability, Tolerance, Phe Levels and Growth

PKU explore France is an exploratory study to evaluate the acceptability of PKU explore, a food for special medical purposes, for use in the dietary management of phenylketonuria in infants from 6 months to 3 years of age, assessing participant adherence, GI tolerance, phe levels, growth and product palatability.

Study Overview

• Status: Active, not recruiting
• Conditions: Phenylketonurias
• Intervention / Treatment: Dietary supplement: PKU explore

Detailed Description

The study product, PKU explore, is a concentrated, spoonable, protein substitute food for special medical purposes, used in the dietary management of PKU. The recommended amount will be determined by a dietitian or clinician and the product used is unflavoured.

PKU explore France is an exploratory study to evaluate the gastrointestinal tolerance, palatability, and participant adherence over a four-week period in patients with PKU that follow an appropriate restricted therapeutic diet. This will be followed by a maximum 24-month follow-up period monitoring growth as per routine standard of care.

Up to 10 participants will be recruited at a single centre in France.

For 28 consecutive days, up to 10 participants, aged 6 months to 3 years inclusive, take their usual restricted therapeutic diet, either replacing their usual second-stage, concentrated protein substitute with PKU explore or commencing PKU explore and gradually reducing their Phe-free formula for infants.

Their parent/guardian complete a daily questionnaire on adherence and tolerance, and a final questionnaire on usage and acceptability.

• Study Type: Interventional
• Enrollment (Actual): 7
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Binyam Tesfaye; Phone Number: +44 (0) 151 709 9020; Email: Binyam.Tesfaye@Vitaflo.co.uk
• Study Contact Backup: Name: Kirsten Imbert; Email: Contact@Kirsten-Clinical-Research.com

Study Locations

• France
  • Hauts-de-France
    • Lille, Hauts-de-France, France, 59037
      • Hopital Jeanne de Flandre

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 months to 3 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• A diagnosis of PKU on new-born screening requiring a low protein diet and Phe-free L-amino acid protein substitute.
• Aged between 6 months and 3 years (inclusive at screening).
• Already taking part of their protein substitute in a spoonable form OR is at the stage in their PKU management when a second stage spoonable protein substitute is recommended to commence.
• Well-controlled PKU, evidenced by the latest three routine blood spots being within the acceptable range, in the investigator's opinion.
• Able to comply with the study protocol and take the study product, according to the opinion of the investigator.
• Willingly given, written, informed consent from parents/guardian.

Exclusion Criteria:

• Diagnosis of persistent hyperphenylalaninaemia, or mild PKU not requiring dietary intervention with a low protein diet and Phe-free L-amino acid supplements.
• Diagnosis of a concurrent condition which may adversely affect developmental progression and feeding ability.
• Known milk or fish allergy/intolerance.
• Patients who are currently participating in, plan to participate in or have participated in an interventional investigational drug, food or medical device trial within 30 days prior to the screening visit.
• Existing significant GI issues which may affect compliance with the study protocol, according to the opinion of the investigator.
• Any medical conditions precluding the study intervention, which in the opinion of the investigator may impact on metabolic control during the study period.
• 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).
• Where applicable, patients NOT covered by Health Insurance System and/or not in compliance with the recommendations of National Law in force.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Supportive Care
• 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 Explore; PKU Explore will be prescribed by the study dietitian based on the patient's individual requirement.	Dietary supplement: PKU explore; For 28 consecutive days, participants will take their usual restricted therapeutic diet, either: - Replacing their usual second stage concentrated protein substitute with PKU explore OR - Commencing PKU explore and gradually reducing their Phe-free formula for infants.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adherence to the recommended amount of study product	Quantitative assessments from subject questionnaires that allow evaluation of compliance with the study product (i.e. actual versus prescribed intake), measured in grams consumed per day.	Days 1 - 28
Product palatability rated on a Likert scale by the patient after 28 days	Patient assessment of study product's palatability using a Likert scale. At the end of the 28-day acceptability phase, participants/parents/guardians will be required to complete a Product Acceptability Questionnaire to record perceptions about: the appearance, smell, taste, aftertaste, texture, packaging/presentation of the product, also the ease of preparation and administration. These will be on a 5-point Likert scale as Loved it, Liked it, Neither liked nor disliked it, Didn't like it and Really didn't like it.	Day 28
Gastrointestinal tolerance daily diary as reported by the patient	Qualitative assessments from subject questionnaires to allow evaluation of any self-reported gastrointestinal symptoms during the study period.	Days 1 - 28
Change in Phe levels	Analysis of results from routine dried blood spots. Change in Phe level at diagnosis and at different timepoints measured as µmol/l.	Previous three routine results prior to Visit 1, Visit 1 (Day 0), Week 1, Week 2, Week 3, Week 4 and Visit 2 (Day 28)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Evaluation of growth for a maximum of two years follow up period	Height/length, weight and head circumference measurements will be combined to complete the growth chart at each visit. All recorded weights, lengths, and head circumferences will be plotted on a Croissance Somatique Des Garçons/Filles De La Naissance A 3 Ans Chart. The units of measurement are height/length in centimeters, weight in kilograms, and head circumference in centimeters.	Visit 1 (day 0), Visit 2 (Day 28) and Every 4 months for a max. 24 Months' Followup
Product adherence for a maximum of two years follow up period by measuring the quantity of product intake	Measurement of the quantity of product intake. Quantitative assessments from subject questionnaires that allow evaluation of compliance with the study product, i.e. actual versus prescribed intake. Weight of powder prescribed per 24 hours in g (grams) and the number of feeds prescribed per 24 hours (feeds) will be recorded.	days 1 - 28
Product adherence for a maximum of two years follow up period by measuring the Phe levels	Measurement of phe levels in µmol/l throughout the evaluation period. The latest Phe level results will be recorded at each visit as µmol/l and compared.	Visit 1 (Day 0), Week 1, Week 2, Week 3, Week 4, Visit 2 (Day 28) and every 4 months for a max. 24 Months' Follow-up period

Collaborators and Investigators

• Sponsor: Vitaflo International, Ltd
• Investigators: Principal Investigator: Karine Mention, CHU Lille

Study record dates

Study Major Dates

• Study Start (Actual): May 18, 2022
• Primary Completion (Estimated): December 31, 2024
• Study Completion (Estimated): May 31, 2025

Study Registration Dates

• First Submitted: May 11, 2021
• First Submitted That Met QC Criteria: May 18, 2021
• First Posted (Actual): May 24, 2021

Study Record Updates

• Last Update Posted (Actual): February 15, 2024
• Last Update Submitted That Met QC Criteria: February 14, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Keywords: France; Phenylketonuria; PKU; Explore
• 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-W-PKUExp-2018-09-03; 2021-A00276-35 (Other Identifier: REC)

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