Evaluation of PKU Explore France

NCT ID: NCT04898829

Last Updated: 2025-11-28

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 7 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-05-18
• Study Completion Date: 2025-10-31

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.

Conditions

Phenylketonurias

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: SUPPORTIVE_CARE
• Blinding Strategy: NONE

Study Groups

PKU Explore

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

Group Type: EXPERIMENTAL

PKU explore

Intervention Type: 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.

Interventions

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.

Intervention Type: DIETARY_SUPPLEMENT

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.

• Minimum Eligible Age: 6 Months
• Maximum Eligible Age: 3 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Vitaflo International, Ltd

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Karine Mention

Role: PRINCIPAL_INVESTIGATOR

CHU Lille

Locations

Hôpital Jeanne de Flandre

Lille, Hauts-de-France, France

Countries

France

Other Identifiers

2021-A00276-35

Identifier Type: OTHER

Identifier Source: secondary_id

MCT-W-PKUExp-2018-09-03

Identifier Type: -

Identifier Source: org_study_id