PhenylketonuriasNCT04375592
Acceptability and Tolerance of a Ready-to-use Protein Substitute in Tablet Form for the Dietary Management of Phenylketonuria
An observational study of Phenylketonurias and Hyperphenylalaninaemia, run by metaX Institut fuer Diatetik GmbH at 1 site in United Kingdom.
Trial summary
The purpose of this prospective, observational study is to evaluate the tolerability and acceptability of phenylalanine-free protein substitute tablets for young children with PKU aged of 7 years or older. Read more about this study
- Who is it for?
- Ages 7 to 18 years
- Conditions the study is for
- Phenylketonurias, Hyperphenylalaninaemia, Tetrahydrobiopterin Deficiency
- What is being tested?
- Phenylalanine-free protein substitute in tablet form (XPhe minis)
- Type of treatment
- Pill or oral medicine
- How many people
- 10 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 1 Feb 2021to 10 May 2021
- Phase
- Observational
- 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 7 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: 10 children with PKU
Who may be able to join
- Diagnosis of PKU or PKU variant requiring a phenylalanine-free protein substitute.
- Subjects who are already taking a phenylalanine-free protein substitute and are willing to try the study product for 7 days.
- Children aged 7 years and over.
- Written informed consent obtained from parental caregiver.
Who cannot join
- Presence of serious concurrent illness
- Lead Dietitian's uncertainty about the willingness or ability of the patient to comply with the protocol requirements
- Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study.
- Any children having taken antibiotics over the previous 2 weeks leading up to the study.
- Children less than 7 years of age.
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
Birmingham Children´s Hospital
Birmingham, Wales, B4 6NH
About this study
Official title: XPhe Minis - Acceptability and Tolerance Market Research
The purpose of this prospective, observational study is to evaluate the tolerability and acceptability of phenylalanine-free protein substitute tablets for young children with PKU aged of 7 years or older.
Read the detailed description
The principle treatment for children with phenylketonuria (PKU) is a low protein diet. Part of this treatment requires the administration of a phenylalanine-free protein substitute in order to meet basic protein requirements for normal growth and development. Several brands of phenylalanine-free protein substitutes are already available in various forms - liquid, semi solid, tablet. However, compliance with taking protein substitutes continues to be a challenge. As a low protein diet is recommended for life, long term compliance is a growing concern. As a result, improving the choice in terms of product type may aid compliance. XPhe minis are phenylalanine-free protein substitute tablets enriched with vitamins, minerals and trace elements designed for children and adults with PKU. It is anticipated that they will be a suitable alternative choice for PKU patients, broadening the variety of protein substitutes they can choose from in order to suit their lifestyle and preferences.
This is a prospective, observational tolerance study in 10 children with PKU. Subjects who are currently taking a second stage protein substitute will be recruited for a 7-day trial, taking the ready-to-use protein substitute tablets to evaluate the tolerability and acceptability of the study product. Therefore, subjects will replace some or all of their usual protein substitute with the new product.
During the 7-days trial subjects or caregivers will be asked to complete a daily questionnaire recording information on: • Usage and compliance • Ease of use and any issues with administration • Any gastro-intestinal side-effects.
A questionnaire will also be completed at the beginning and end of the study that will consider perceptions about taste, appearance, smell, ease of administration; how it is taken; and any other problems or symptoms.
The amount of tablets prescribed will be calculated to provide the same amount of protein as their usual protein substitute.
Subjects will continue to have weekly finger-prick blood tests as is routine in PKU. The results whilst on the study product will be compared with results whilst on their usual protein substitute.
10 children with PKU will be recruited. When an appropriate subject has been identified, a study information sheet will be sent to the subject or parents/caregivers. They will be invited to request further information about the study if they wish by contacting the Lead Dietitian. Recruitment of each patient will be by written informed consent, which will be completed by the parents/primary caregivers and taken by the Lead Dietitian. Children will also complete an assent/consent form and will receive an information sheet, if considered appropriate for their level of understanding.
- Being tested
- Phenylalanine-free protein substitute in tablet form (XPhe minis)
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Phenylalanine-free protein substitute in tablet form (XPhe minis)Dietary supplement
Intervention is a Phenylalanine-free protein substitute in tablet form. Subjects who currently take a concentrated second stage phenylalanine-free protein substitute will be recruited. Subjects will take the study product for 7 days and daily questionnaires will be completed. Subjects will replace some or all of their usual protein substitute with the new product. The amount of study product prescribed will be calculated to provide the same amount of protein as their usual protein substitute. Daily acceptability and tolerance questionnaires will record information on ease of preparation and administration; how it is taken; and any problems or gastrointestinal effects. Additional questions at the beginning and end of the study will record information on taste, appearance, smell, presentation and packaging of the product. Routine weekly finger prick blood spots will be collected and analysed for phenylalanine and tyrosine as usual.
What is being measured
The primary outcome is the main question the study is designed to answer.
Daily compliance
Compliance, with currently prescribed protein substitute will be assessed at the beginning of the 7-days trial. Usage and compliance with the study product will subsequently assessed daily from days 1-7 using standardised questionnaires, where patients document the amount of consumed study product vs the prescribed doses. Compliance
Measured: 7 days
Secondary outcomes (4)
Treatment-Emergent tolerability
Gastro-intestinal side effects (including diarrhoea, constipation, bloating or abdominal distension, nausea, vomiting, burping, flatulence or regurgitation and abdominal discomfort or pain) will be assessed daily via questionnaire using the scale (none today, mild, moderate or severe / really troublesome).
Measured: 7 days
Patient Acceptability
Acceptability (ease of use and palatability) will be assessed via questionnaire daily during the study period and at the end of the study via questionnaire .
Measured: 7 days
Metabolic control
Weekly routine finger prick blood spots will be collected and analysed for phenylalanine (micromole per Liter) and tyrosine (micromole per Liter) as is usual clinical practice.
Measured: 2 days
Incidence of study product emergent events.
All adverse events will be recorded throughout the study (as and when they occur) and after study completion, an average of 1 year. As the study product is already commercially available occurence of any adverse event is unlikely.
Measured: Through study completion an average of 1year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 May 2020
- Study start1 Feb 2021
- Primary completion1 May 2021
- Study completion10 May 2021
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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- metaX Institut fuer Diatetik GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Birmingham Children's Hospital
- Responsible party
- Sponsor
- Investigators
- Anita MacDonald, Professor, Principal investigator, Birmingham Children´s Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04375592
- Sponsor's protocol ID
- XPheminisCT01
- Status last verified
- Mar 2024
- First submitted
- 21 Apr 2020
- Last updated
- 4 Mar 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: No individual patient data will be shared with other researchers.
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- XPhe Minis - Acceptability and Tolerance Market Research
- ClinicalTrials.gov ID
- NCT04375592
- Acronym / sponsor ID
- XPheminisCT01
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- metaX Institut fuer Diatetik GmbHIndustry (pharma, biotech, devices)
- Collaborators
- Birmingham Children's Hospital
- Study start
- 1 Feb 2021 (actual)
- Primary completion
- 1 May 2021 (actual)
- Study completion
- 10 May 2021 (actual)
- First posted
- 5 May 2020 (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.
The purpose of this prospective, observational study is to evaluate the tolerability and acceptability of phenylalanine-free protein substitute tablets for young children with PKU aged of 7 years or older.
Detailed description
The principle treatment for children with phenylketonuria (PKU) is a low protein diet. Part of this treatment requires the administration of a phenylalanine-free protein substitute in order to meet basic protein requirements for normal growth and development. Several brands of phenylalanine-free protein substitutes are already available in various forms - liquid, semi solid, tablet. However, compliance with taking protein substitutes continues to be a challenge. As a low protein diet is recommended for life, long term compliance is a growing concern. As a result, improving the choice in terms of product type may aid compliance. XPhe minis are phenylalanine-free protein substitute tablets enriched with vitamins, minerals and trace elements designed for children and adults with PKU. It is anticipated that they will be a suitable alternative choice for PKU patients, broadening the variety of protein substitutes they can choose from in order to suit their lifestyle and preferences.
This is a prospective, observational tolerance study in 10 children with PKU. Subjects who are currently taking a second stage protein substitute will be recruited for a 7-day trial, taking the ready-to-use protein substitute tablets to evaluate the tolerability and acceptability of the study product. Therefore, subjects will replace some or all of their usual protein substitute with the new product.
During the 7-days trial subjects or caregivers will be asked to complete a daily questionnaire recording information on: • Usage and compliance • Ease of use and any issues with administration • Any gastro-intestinal side-effects.
A questionnaire will also be completed at the beginning and end of the study that will consider perceptions about taste, appearance, smell, ease of administration; how it is taken; and any other problems or symptoms.
The amount of tablets prescribed will be calculated to provide the same amount of protein as their usual protein substitute.
Subjects will continue to have weekly finger-prick blood tests as is routine in PKU. The results whilst on the study product will be compared with results whilst on their usual protein substitute.
10 children with PKU will be recruited. When an appropriate subject has been identified, a study information sheet will be sent to the subject or parents/caregivers. They will be invited to request further information about the study if they wish by contacting the Lead Dietitian. Recruitment of each patient will be by written informed consent, which will be completed by the parents/primary caregivers and taken by the Lead Dietitian. Children will also complete an assent/consent form and will receive an information sheet, if considered appropriate for their level of understanding.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 7 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- 10 children with PKU
Key inclusion criteria (4 listed)
- Diagnosis of PKU or PKU variant requiring a phenylalanine-free protein substitute.
- Subjects who are already taking a phenylalanine-free protein substitute and are willing to try the study product for 7 days.
- Children aged 7 years and over.
- Written informed consent obtained from parental caregiver.
Key exclusion criteria (5 listed)
- Presence of serious concurrent illness
- Lead Dietitian's uncertainty about the willingness or ability of the patient to comply with the protocol requirements
- Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study.
- Any children having taken antibiotics over the previous 2 weeks leading up to the study.
- Children less than 7 years of age.
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 |
|---|---|---|
| Phenylalanine-free protein substitute in tablet form (XPhe minis)Intervention is a Phenylalanine-free protein substitute in tablet form. Subjects who currently take a concentrated second stage phenylalanine-free protein substitute will be recruited. Subjects will take the study product for 7 days and daily questionnaires will be completed. Subjects will replace some or all of their usual protein substitute with the new product. The amount of study product prescribed will be calculated to provide the same amount of protein as their usual protein substitute. Daily acceptability and tolerance questionnaires will record information on ease of preparation and administration; how it is taken; and any problems or gastrointestinal effects. Additional questions at the beginning and end of the study will record information on taste, appearance, smell, presentation and packaging of the product. Routine weekly finger prick blood spots will be collected and analysed for phenylalanine and tyrosine as usual. | Dietary supplement | – |
Primary outcome
Daily compliance
Time frame: 7 days
Compliance, with currently prescribed protein substitute will be assessed at the beginning of the 7-days trial. Usage and compliance with the study product will subsequently assessed daily from days 1-7 using standardised questionnaires, where patients document the amount of consumed study product vs the prescribed doses. Compliance
4 secondary outcomes
Treatment-Emergent tolerability
Time frame: 7 days
Patient Acceptability
Time frame: 7 days
Metabolic control
Time frame: 2 days
Incidence of study product emergent events.
Time frame: Through study completion an average of 1year
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 4 Mar 2024.
- Individual participant dataSharing planned: No. No individual patient data will be shared with other researchers.
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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, Professor principal investigator
Birmingham Children´s Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Phenylketonurias and Hyperphenylalaninaemia, run by metaX Institut fuer Diatetik GmbH at 1 site 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 4 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 4 Mar 2024. 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 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?
Daily compliance (time frame: 7 days). 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 4 Mar 2024.