Clinical trial NCT04898829: 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
Overall status: ACTIVE_NOT_RECRUITING
Phase: NA
Conditions: Phenylketonurias
Keywords: Phenylketonuria
PKU
Explore
France
Lead sponsor: Vitaflo International, Ltd
Sponsor class: INDUSTRY
First posted: 2021-05-24
Enrollment: 7
Sex: ALL
Minimum age (years): 6
Maximum age (years): 3
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: SUPPORTIVE_CARE

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.

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.

Primary outcomes:
measure: Adherence to the recommended amount of study product
timeFrame: Days 1 - 28
measure: Product palatability rated on a Likert scale by the patient after 28 days
timeFrame: Day 28
measure: Gastrointestinal tolerance daily diary as reported by the patient
timeFrame: Days 1 - 28
measure: Change in Phe levels
timeFrame: 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:
measure: Evaluation of growth for a minimum of two years follow up period
timeFrame: Visit 1 (day 0), Visit 2 (Day 28) and Every 4 months for a max. 24 Months' Followup
measure: Product adherence for a minimum of two years follow up period by measuring the quantity of product intake
timeFrame: days 1 - 28
measure: Product adherence for a minimum of two years follow up period by measuring the Phe levels
timeFrame: 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
Other outcomes:
Locations:
city: Lille
country: France
facility: Hôpital Jeanne de Flandre
state: Hauts-de-France
Study officials:
affiliation: CHU Lille
name: Karine Mention
role: PRINCIPAL_INVESTIGATOR
References: