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Evaluation of PKU Explore France

Primary Purpose

Phenylketonurias

Status

Recruiting

Phase

Not Applicable

Locations

France

Study Type

Interventional

Intervention

PKU explore

About this trial

This is an interventional supportive care trial for Phenylketonurias focused on measuring Phenylketonuria, PKU, Explore, France

Eligibility Criteria

6 Months - 3 Years (Child)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Hôpital Jeanne de FlandreRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

PKU Explore

Arm Description

PKU Explore will be prescribed by the study dietitian based on the patient's individual requirement.

Outcomes

Primary Outcome Measures

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.

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.

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.

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.

Secondary Outcome Measures

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.

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.

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.

Full Information

NCT ID

NCT04898829

First Posted

May 11, 2021

Last Updated

August 18, 2023

Sponsor

Vitaflo International, Ltd

1. Study Identification

Unique Protocol Identification Number

NCT04898829

Brief Title

Evaluation of PKU Explore France

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

August 2023

Overall Recruitment Status

Recruiting

Study Start Date

May 18, 2022 (Actual)

Primary Completion Date

December 31, 2023 (Anticipated)

Study Completion Date

May 31, 2025 (Anticipated)

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

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief Summary

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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Phenylketonurias

Keywords

Phenylketonuria, PKU, Explore, France

7. Study Design

Primary Purpose

Supportive Care

Study Phase

Not Applicable

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

PKU Explore

Arm Type

Experimental

Arm Description

PKU Explore will be prescribed by the study dietitian based on the patient's individual requirement.

Intervention Type

Dietary Supplement

Intervention Name(s)

PKU explore

Intervention Description

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.

Primary Outcome Measure Information:

Title

Adherence to the recommended amount of study product

Description

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.

Time Frame

Days 1 - 28

Title

Product palatability rated on a Likert scale by the patient after 28 days

Description

Time Frame

Day 28

Title

Gastrointestinal tolerance daily diary as reported by the patient

Description

Qualitative assessments from subject questionnaires to allow evaluation of any self-reported gastrointestinal symptoms during the study period.

Time Frame

Days 1 - 28

Title

Change in Phe levels

Description

Analysis of results from routine dried blood spots. Change in Phe level at diagnosis and at different timepoints measured as µmol/l.

Time Frame

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 Measure Information:

Title

Evaluation of growth for a maximum of two years follow up period

Description

Time Frame

Visit 1 (day 0), Visit 2 (Day 28) and Every 4 months for a max. 24 Months' Followup

Title

Product adherence for a maximum of two years follow up period by measuring the quantity of product intake

Description

Time Frame

days 1 - 28

Title

Product adherence for a maximum of two years follow up period by measuring the Phe levels

Description

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.

Time Frame

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

10. Eligibility

Sex

All

Minimum Age & Unit of Time

6 Months

Maximum Age & Unit of Time

3 Years

Accepts Healthy Volunteers

Eligibility Criteria

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.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Binyam Tesfaye

Phone

+44 (0) 151 709 9020

Binyam.Tesfaye@Vitaflo.co.uk

First Name & Middle Initial & Last Name or Official Title & Degree

Kirsten Imbert

Contact@Kirsten-Clinical-Research.com

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Karine Mention

Organizational Affiliation

CHU Lille

Official's Role

Principal Investigator

Facility Information:

Facility Name

Hôpital Jeanne de Flandre

City

Lille

State/Province

Hauts-de-France

ZIP/Postal Code

59037

Country

France

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Karine Mention

Karine.Mention@CHRU-Lille.Fr

First Name & Middle Initial & Last Name & Degree

Dries Dobbelaere

Dries.Dobbelaere@CHRU-Lille.Fr

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

Evaluation of PKU Explore France

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