PhenylketonuriasNCT04898829
Evaluation of PKU Explore France
A study testing PKU explore in people with Phenylketonurias, run by Vitaflo International, Ltd at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 months to 3 years
- Conditions the study is for
- Phenylketonurias
- What is being tested?
- PKU explore
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteFrance
- Study dates
- 18 May 2022to 31 Oct 2025
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 3 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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
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.
Read the 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
- Being tested
- PKU explore
- Keywords
- Phenylketonuria, PKU, Explore, France
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PKU exploreDietary 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.
| Study group | Role | What they receive |
|---|---|---|
| PKU Explore | Experimental | PKU Explore will be prescribed by the study dietitian based on the patient's individual requirement. Receives: Dietary Supplement: PKU explore |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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
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.
Measured: 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
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.
Measured: days 1 - 28
Product adherence for a minimum 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.
Measured: 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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 May 2021
- Study start18 May 2022
- Primary completion31 Oct 2025
- Study completion31 Oct 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PhenylketonuriasStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vitaflo International, Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Karine Mention, Principal investigator, CHU Lille
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04898829
- Sponsor's protocol ID
- MCT-W-PKUExp-2018-09-03
- Other IDs
- 2021-A00276-35
- Status last verified
- Nov 2025
- First submitted
- 11 May 2021
- Last updated
- 28 Nov 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04898829
- Acronym / sponsor ID
- MCT-W-PKUExp-2018-09-03
- Conditions
- Phenylketonurias
- Keywords
- Phenylketonuria, PKU, Explore, France
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Vitaflo International, LtdIndustry (pharma, biotech, devices)
- Study start
- 18 May 2022 (actual)
- Primary completion
- 31 Oct 2025 (actual)
- Study completion
- 31 Oct 2025 (actual)
- First posted
- 24 May 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PKU Explore | Experimental | Dietary Supplement: PKU explorePKU Explore will be prescribed by the study dietitian based on the patient's individual requirement. |
Who may qualify
- Ages
- 6 months to 3 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PKU exploreFor 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. | Dietary supplement | PKU Explore |
Primary outcome
Adherence to the recommended amount of study product
Time frame: Days 1 - 28
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
Time frame: Day 28
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
Time frame: Days 1 - 28
Qualitative assessments from subject questionnaires to allow evaluation of any self-reported gastrointestinal symptoms during the study period.
Change in Phe levels
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)
Analysis of results from routine dried blood spots. Change in Phe level at diagnosis and at different timepoints measured as µmol/l.
3 secondary outcomes
Evaluation of growth for a minimum of two years follow up period
Time frame: 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
Time frame: days 1 - 28
Product adherence for a minimum of two years follow up period by measuring the Phe levels
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Nov 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Karine Mention principal investigator
CHU Lille
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing PKU explore in people with Phenylketonurias, run by Vitaflo International, Ltd at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adherence to the recommended amount of study product (time frame: Days 1 - 28). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Nov 2025.