Evaluation of PKU Explore France (NCT04898829) | Clinical Trial Compass
CompletedNot Applicable
Evaluation of PKU Explore France
France7 participantsStarted 2022-05-18
Plain-language 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.
Who can participate
Age range
6 Months – 3 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Adherence to the recommended amount of study product
Timeframe: Days 1 - 28
2
Product palatability rated on a Likert scale by the patient after 28 days
Timeframe: Day 28
3
Gastrointestinal tolerance daily diary as reported by the patient
Timeframe: Days 1 - 28
4
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)