Evaluation of PKU Explore France

NCT04898829 | Completed

• 2 other identifiers: MCT-W-PKUExp-2018-09-032021-A00276-35
• Study Type: interventional
• Target: 7
• Locations: 1 country
• Sites: 1

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.

Conditions

Phenylketonurias

Keywords

Phenylketonuria; PKU; Explore; France

Outcome Measures

Primary Outcomes (4)

• 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 Outcomes (3)

• Evaluation of growth for a minimum of two years follow up period

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

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

  days 1 - 28

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

  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

Study Arms (1)

PKU Explore

EXPERIMENTAL

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

Dietary Supplement: PKU explore

Interventions

PKU explore DIETARY_SUPPLEMENT

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.

PKU Explore

Eligibility Criteria

• Age: 6 Months - 3 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Vitaflo International, Ltd: lead

Study Sites (1)

Hôpital Jeanne de Flandre

Lille, Hauts-de-France, 59037, France

Location

MeSH Terms

Conditions

Phenylketonurias

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Karine Mention

  CHU Lille

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 11, 2021
• First Posted: May 24, 2021
• Study Start: May 18, 2022
• Primary Completion: October 31, 2025
• Study Completion: October 31, 2025
• Last Updated: November 28, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)