PhenylketonuriaPKU StartNCT03058848
Evaluation of PKU Start
A study testing PKU Start 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 Start, a new Phe free protein substitute for the dietary management of PKU in infants from birth. Read more about this study
- Who is it for?
- Ages 4 weeks to 2 years
- Conditions the study is for
- Phenylketonuria, Inborn Errors of Metabolism
- What is being tested?
- PKU Start
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 3 sitesUnited Kingdom
- Study dates
- 6 Mar 2017to 15 Sep 2017
- 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 4 weeks to 2 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- A diagnosis of classical or severe PKU on new-born screening (For the purposes of this study, 'severe' is defined as phe concentrations between 120 and 600 µmol/L at diagnosis)
- Taking a minimum of one (1) feed of a Phe-free infant formula
- A minimum period of four (4) weeks from the time of diagnosis to initial approach to parents
Who cannot join
- Diagnosed with mild PKU or hyperphenylalaninaemia (For the purposes of this study, 'mild' is defined as phe concentrations between 120 and 600 µmol/L at diagnosis)
- Diagnosis of a congenital condition
Trial locations and contacts
3 sites in 1 country.
United Kingdom3 sites
Birmingham Children's Hospital NHS Foundation Trust
Birmingham, West Midlands, B4 6NH
Bradford Teaching Hospitals NHS Foundation Trust
Bradford, West Yorkshire, BD5 0NA
NHS Greater Glasgow and Clyde
Glasgow, Lanarkshire, G51 4TF
About this study
Official title: A Study to Evaluate the Acceptability of a New Phenylalanine Free Infant Formula for Use in the Dietary Management of Phenylketonuria in Infants From Birth to 2 Year of Age With Regard to Product Tolerance and Adherence.
To evaluate the acceptability, tolerance and effect on metabolic control of PKU Start, a new Phe free protein substitute for the dietary management of PKU in infants from birth.
Read the detailed description
This is an assessment of ten (10) infants who require a protein restricted diet that is low in Phe. Infants who routinely use a Phe free infant formula as part of their dietary therapy will be recruited for a 28-day assessment of PKU Start, to evaluate tolerance and acceptability.
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 Start based on individual requirements. Parents/carers will be asked to substitute their usual Phe-free infant formula with PKU Start for one (1) month. The sponsor will supply PKU Start free of charge.
Prior to starting PKU Start, parents/carers will be asked to record information about the infant's usual GI tolerance and feeding pattern for a period of three (3) days, to allow for comparison between their existing formula and PKU Start.
They will be asked to record information about the following:
Stools Vomiting and Spit-up Feed / Fluid Intake and Compliance Phenylalanine Levels Final Evaluation about the presentation of the product, ease of preparation and how PKU Start flowed through the teat of a bottle.
- Conditions
- Phenylketonuria, Inborn Errors of Metabolism
- Being tested
- PKU Start
- 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 StartDietary supplement
PKU Start is a powdered, phenylalanine-free, infant formula, containing essential and non-essential amino acids, carbohydrate, fat, vitamins, minerals, trace elements and long chain polyunsaturated fatty acids (LCPs); Arachidonic acid (AA) and Docosahexaenoic acid (DHA). It is suitable for use from birth.
The recommended amount of the product for each participant will be determined and prescribed by a dietitian.
| Study group | Role | What they receive |
|---|---|---|
| Consumption of PKU Start | Experimental | Daily feed, substituting the participant's normal phe-free formula for PKU Start. Receives: Dietary Supplement: PKU Start |
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
Qualitative assessments from subject questionnaires that allow evaluation of the gastro-intestinal tolerance of the study product.
Measured: Days 1-28
Ease of use questionnaire
Qualitative assessment from subject questionnaire that allows evaluation of the ease of use of the study product.
Measured: Day 29
Daily 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 posted23 Feb 2017
- Study start6 Mar 2017
- Primary completion15 Sep 2017
- Study completion15 Sep 2017
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
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vitaflo International, Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Anita MacDonald, Principal investigator, Birmingham Women's and Children's NHS Foundation Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03058848
- Sponsor's protocol ID
- BCH-PKUSTART-032016-29
- Other IDs
- 209920, 17/NW/0035
- Acronym
- PKU Start
- Status last verified
- Feb 2024
- First submitted
- 16 Feb 2017
- Last updated
- 16 Feb 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
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 a New Phenylalanine Free Infant Formula for Use in the Dietary Management of Phenylketonuria in Infants From Birth to 2 Year of Age With Regard to Product Tolerance and Adherence.
- ClinicalTrials.gov ID
- NCT03058848
- Acronym / sponsor ID
- PKU Start / BCH-PKUSTART-032016-29
- Conditions
- Phenylketonuria, Inborn Errors of Metabolism
- Keywords
- Phenylketonuria, PKU, Metabolism, Metabolic, Control, Acceptability
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Vitaflo International, LtdIndustry (pharma, biotech, devices)
- Study start
- 6 Mar 2017 (actual)
- Primary completion
- 15 Sep 2017 (actual)
- Study completion
- 15 Sep 2017 (actual)
- First posted
- 23 Feb 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 Start, a new Phe free protein substitute for the dietary management of PKU in infants from birth.
Detailed description
This is an assessment of ten (10) infants who require a protein restricted diet that is low in Phe. Infants who routinely use a Phe free infant formula as part of their dietary therapy will be recruited for a 28-day assessment of PKU Start, to evaluate tolerance and acceptability.
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 Start based on individual requirements. Parents/carers will be asked to substitute their usual Phe-free infant formula with PKU Start for one (1) month. The sponsor will supply PKU Start free of charge.
Prior to starting PKU Start, parents/carers will be asked to record information about the infant's usual GI tolerance and feeding pattern for a period of three (3) days, to allow for comparison between their existing formula and PKU Start.
They will be asked to record information about the following:
Stools Vomiting and Spit-up Feed / Fluid Intake and Compliance Phenylalanine Levels Final Evaluation about the presentation of the product, ease of preparation and how PKU Start flowed through the teat of a bottle.
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 Start | Experimental | Dietary Supplement: PKU StartDaily feed, substituting the participant's normal phe-free formula for PKU Start. |
Who may qualify
- Ages
- 4 weeks to 2 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- A diagnosis of classical or severe PKU on new-born screening (For the purposes of this study, 'severe' is defined as phe concentrations between 120 and 600 µmol/L at diagnosis)
- Taking a minimum of one (1) feed of a Phe-free infant formula
- A minimum period of four (4) weeks from the time of diagnosis to initial approach to parents
Key exclusion criteria (2 listed)
- Diagnosed with mild PKU or hyperphenylalaninaemia (For the purposes of this study, 'mild' is defined as phe concentrations between 120 and 600 µmol/L at diagnosis)
- Diagnosis of a congenital condition
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 StartPKU Start is a powdered, phenylalanine-free, infant formula, containing essential and non-essential amino acids, carbohydrate, fat, vitamins, minerals, trace elements and long chain polyunsaturated fatty acids (LCPs); Arachidonic acid (AA) and Docosahexaenoic acid (DHA). It is suitable for use from birth. The recommended amount of the product for each participant will be determined and prescribed by a dietitian. | Dietary supplement | Consumption of PKU Start |
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
Time frame: Days 1-28
Qualitative assessments from subject questionnaires that allow evaluation of 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.
Daily 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: Undecided
Related trials for Phenylketonuria
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Not yet recruiting
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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 Start 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 3 inclusion and 2 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.