PhenylketonuriaPKU ExploreNCT03168399
Evaluation of PKU Explore
A study testing PKU Explore in people with Phenylketonuria and Inborn Errors of Metabolism, run by Vitaflo International, Ltd at 3 sites in United Kingdom.
Trial summary
To evaluate the acceptability, tolerance and effect on metabolic control of PKU Explore, a renovated Phe free protein substitute for the dietary management of PKU in children from 6 months to 5 years. Read more about this study
- Who is it for?
- Ages 6 months to 5 years
- Conditions the study is for
- Phenylketonuria, Inborn Errors of Metabolism
- What is being tested?
- PKU Explore
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 3 sitesUnited Kingdom
- Study dates
- 8 Jun 2017to 15 Mar 2018
- 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 5 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 supplements.
- Aged between 6 months and 5 years.
- Already taking a second stage concentrated protein substitute as part of their PKU management OR is at the stage in their PKU management when a second stage concentrated protein substitute is recommended to commence.
- Willingly given, written, informed consent from parent/guardian.
- Willingly given, written assent (if appropriate).
Who cannot join
- Diagnosis of persistent hyperphenylalaninaemia, or mild PKU not requiring dietary treatment 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.
- Participation in any other clinical trial/acceptability study.
- Any serious medical precluding the study intervention.
- 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).
Trial locations and contacts
3 sites in 1 country.
United Kingdom3 sites
Bradford Teaching Hospitals NHS Foundation Trust
Bradford, West Yorkshire, BD5 0NA
Birmingham Women's and Children's NHS Foundation Trust
Burringham, West Midlands, B4 6NH
NHS Greater Glasgow and Clyde
Glasgow, Lanarkshire, G51 4TF
About this study
Official title: A Study to Evaluate the Acceptability of PKU Explore, a Renovated Phenylalanine Free Second Stage Protein Substitute for Use in the Dietary Management of Phenylketonuria in Infants From 6 Months to 5 Years of Age With Regard to Product Tolerance and Adherence.
To evaluate the acceptability, tolerance and effect on metabolic control of PKU Explore, a renovated Phe free protein substitute for the dietary management of PKU in children from 6 months to 5 years.
Read the detailed description
This is a prospective, acceptability study to evaluate the gastrointestinal tolerance, palatability and adherence of PKU explore in a four-week period for 15 participants aged between 6 months and 5 years for the dietary management of PKU.
The outcome of this assessment will be used in a submission to the regulatory authorities, Advisory Committee on Borderline Substances (ACBS), for PKU Start to become reimbursable on prescription in the UK.
The participant's dietitian will advise on an appropriate amount of PKU Explore based on individual requirements. Parents/carers will be asked to substitute their usual Phe-free protein substitute with PKU Explore for one (1) month. The sponsor will supply PKU Explore free of charge.
Parents/guardians will be asked to record information about the following:
Daily PKU Explore intake Stools Vomiting and Spit-up Feed / Fluid Intake Compliance Phenylalanine Levels (from their routine blood spot testing)
Final Evaluation about the presentation of the product, ease of preparation and use.
- Conditions
- Phenylketonuria, Inborn Errors of Metabolism
- Being tested
- PKU Explore
- Keywords
- Phenylketonuria, PKU, Metabolism, Metabolic, Control, Acceptability
- Medical subject headings
- Phenylketonurias, Metabolism, Inborn Errors
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PKU ExploreDietary supplement
PKU Explore is an unflavoured, powdered, phenylalanine-free, protein substitute, containing essential and non-essential amino acids, carbohydrate, sugar, vitamins, minerals, trace elements and the long chain polyunsaturated fatty acids (LCPs); arachidonic acid (AA) and docosahexaenoic acid (DHA).
| Study group | Role | What they receive |
|---|---|---|
| Consumption of PKU Explore | Experimental | Daily feed, substituting the participant's normal phe-free protein substitute for PKU Explore. Receives: Dietary Supplement: PKU Explore |
What is being measured
The primary outcome is the main question the study is designed to answer.
Product compliance daily diary
Quantitative assessments from subject questionnaires that allow evaluation of compliance with the study product, i.e. actual versus prescribed intake.
Measured: Days 1-28
GI tolerance daily diary assessing change
Qualitative assessments from subject questionnaires that allow evaluation of any change in the gastro-intestinal tolerance of the study product.
Measured: Days 1-7 and 22-28
Ease of use questionnaire
Qualitative assessment from subject questionnaire that allows evaluation of the ease of use of the study product.
Measured: Day 29
Weekly phenylalanine control
Collection of quantitative data regarding phenylalanine control using routine biochemical testing
Measured: Days 1-28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 May 2017
- Study start8 Jun 2017
- Primary completion15 Mar 2018
- Study completion15 Mar 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PhenylketonuriaStudies 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
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vitaflo International, Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Birmingham Women's and Children's NHS Foundation Trust
- Responsible party
- Sponsor
- Investigators
- Anita MacDonald, Principal investigator, Birmingham Women's and Children's NHS Foundation Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03168399
- Sponsor's protocol ID
- MCT-PKUE-2016-12-05
- Other IDs
- 219119
- Acronym
- PKU Explore
- Status last verified
- Feb 2024
- First submitted
- 24 May 2017
- Last updated
- 16 Feb 2024
- 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 16 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study to Evaluate the Acceptability of PKU Explore, a Renovated Phenylalanine Free Second Stage Protein Substitute for Use in the Dietary Management of Phenylketonuria in Infants From 6 Months to 5 Years of Age With Regard to Product Tolerance and Adherence.
- ClinicalTrials.gov ID
- NCT03168399
- Acronym / sponsor ID
- PKU Explore / MCT-PKUE-2016-12-05
- Conditions
- Phenylketonuria, Inborn Errors of Metabolism
- Keywords
- Phenylketonuria, PKU, Metabolism, Metabolic, Control, Acceptability
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Vitaflo International, LtdIndustry (pharma, biotech, devices)
- Collaborators
- Birmingham Women's and Children's NHS Foundation Trust
- Study start
- 8 Jun 2017 (actual)
- Primary completion
- 15 Mar 2018 (actual)
- Study completion
- 15 Mar 2018 (actual)
- First posted
- 30 May 2017 (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.
To evaluate the acceptability, tolerance and effect on metabolic control of PKU Explore, a renovated Phe free protein substitute for the dietary management of PKU in children from 6 months to 5 years.
Detailed description
This is a prospective, acceptability study to evaluate the gastrointestinal tolerance, palatability and adherence of PKU explore in a four-week period for 15 participants aged between 6 months and 5 years for the dietary management of PKU.
The outcome of this assessment will be used in a submission to the regulatory authorities, Advisory Committee on Borderline Substances (ACBS), for PKU Start to become reimbursable on prescription in the UK.
The participant's dietitian will advise on an appropriate amount of PKU Explore based on individual requirements. Parents/carers will be asked to substitute their usual Phe-free protein substitute with PKU Explore for one (1) month. The sponsor will supply PKU Explore free of charge.
Parents/guardians will be asked to record information about the following:
Daily PKU Explore intake Stools Vomiting and Spit-up Feed / Fluid Intake Compliance Phenylalanine Levels (from their routine blood spot testing)
Final Evaluation about the presentation of the product, ease of preparation and use.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Consumption of PKU Explore | Experimental | Dietary Supplement: PKU ExploreDaily feed, substituting the participant's normal phe-free protein substitute for PKU Explore. |
Who may qualify
- Ages
- 6 months to 5 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- A diagnosis of PKU on new-born screening requiring a low protein diet and Phe-free L-amino acid supplements.
- Aged between 6 months and 5 years.
- Already taking a second stage concentrated protein substitute as part of their PKU management OR is at the stage in their PKU management when a second stage concentrated protein substitute is recommended to commence.
- Willingly given, written, informed consent from parent/guardian.
- Willingly given, written assent (if appropriate).
Key exclusion criteria (5 listed)
- Diagnosis of persistent hyperphenylalaninaemia, or mild PKU not requiring dietary treatment 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.
- Participation in any other clinical trial/acceptability study.
- Any serious medical precluding the study intervention.
- 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).
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 ExplorePKU Explore is an unflavoured, powdered, phenylalanine-free, protein substitute, containing essential and non-essential amino acids, carbohydrate, sugar, vitamins, minerals, trace elements and the long chain polyunsaturated fatty acids (LCPs); arachidonic acid (AA) and docosahexaenoic acid (DHA). | Dietary supplement | Consumption of PKU Explore |
Primary outcome
Product compliance daily diary
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.
GI tolerance daily diary assessing change
Time frame: Days 1-7 and 22-28
Qualitative assessments from subject questionnaires that allow evaluation of any change in the gastro-intestinal tolerance of the study product.
Ease of use questionnaire
Time frame: Day 29
Qualitative assessment from subject questionnaire that allows evaluation of the ease of use of the study product.
Weekly phenylalanine control
Time frame: Days 1-28
Collection of quantitative data regarding phenylalanine control using routine biochemical testing
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 16 Feb 2024.
- 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.
Anita MacDonald principal investigator
Birmingham Women's and Children's NHS Foundation Trust
3 sites 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 Phenylketonuria and Inborn Errors of Metabolism, run by Vitaflo International, Ltd at 3 sites in United Kingdom.
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 5 inclusion and 5 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 16 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United Kingdom. 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?
Product compliance daily diary (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 16 Feb 2024.