TyrosinemiasNCT04196959
Evaluation of TYR Sphere
A study testing TYR sphere in people with Tyrosinemias and Alkaptonuria, run by Vitaflo International, Ltd at 2 sites in United Kingdom.
Trial summary
For 28 days, 20 patients with tyrosinaemia will take TYR sphere as part of the usual dietary management of their condition. Patients will record their adherence to recommended intakes each day, gastrointestinal symptoms (if any) experienced each day of weeks one and four, and the palatability of the product at the end… Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Tyrosinemias, Alkaptonuria, Tyrosinemia, Type I
- What is being tested?
- TYR sphere
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 2 sitesUnited Kingdom
- Study dates
- 1 Oct 2019to 1 Aug 2020
- 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 3 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosed with tyrosinaemia.
- Aged ≥ 3 years.
- In the opinion of the Investigator, can comply with the study protocol and take at least one sachet of the study product per day.
- Willingly given, written, informed consent from patient or parent/guardian.
- Willingly given, written assent (if appropriate).
Who cannot join
- Individuals who are allergic to milk, fish and soya (these allergens are inherent in the study product ingredients).
- Use of additional macro/micronutrient supplements during the study period, unless clinically indicated and prescribed by the Investigator (must be recorded in patient case report form CRF).
- Women who are pregnant / breastfeeding at the start of the study or planning to become pregnant during the study period.
- Individuals who, in the opinion of the investigator, are unable to comply with the requirements of the protocol.
- Any co-morbidity, which, in the opinion of the Investigator, would preclude participation in the study.
- 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 screening visit.
Trial locations and contacts
2 sites in 1 country.
United Kingdom2 sites
Birmingham Children's Hospital
Birmingham, West Midlands, B4 6NH
National Alkaptonuria Centre, Royal Liverpool University Hospital
Liverpool, Merseyside, L7 8XP
About this study
Official title: An Acceptability Study to Evaluate the Adherence, Tolerance and Metabolic Control of Patients With Tyrosinaemia, When Using TYR Sphere (a Food for Special Medical Purposes (FSMP)) as Part of Dietary Management.
For 28 days, 20 patients with tyrosinaemia will take TYR sphere as part of the usual dietary management of their condition. Patients will record their adherence to recommended intakes each day, gastrointestinal symptoms (if any) experienced each day of weeks one and four, and the palatability of the product at the end of the trial. Metabolic control will be evaluated by dried blood spots collected during the Baseline and End of Study visits, and weekly during the 28 days of the study itself.
Trial data will be used in an application to make TYR sphere available via the NHS.
Read the detailed description
TYR sphere is a glycomacropeptide (GMP) based protein substitute for patients with tyrosinaemia.
The Evaluation of TYR sphere study has been designed to generate data required by the United Kingdom's Advisory Committee on Borderline Substances (ACBS), the national regulator for Foods for Special Medical Purposes (FSMP).
The ACBS specify that before a FSMP can be routinely prescribed by the NHS, its acceptability must first be evaluated by a clinical trial in patients for whom it is ultimately designed. The ACBS define acceptability as comprising patient evaluations of the product's palatability, data on their adherence to recommended intakes and their gastrointestinal tolerance of it. TYR sphere is designed for patients of three years of age and upwards. This has informed the trial's age-related inclusion criteria.
Using a mobile phone application, or paper diary form, patients will record their intake of TYR sphere along with the recommended amount on each of the 28 study days. Gastrointestinal tolerance will be recorded each day during weeks one and four, while palatability of TYR sphere will be recorded at the end of the 28 days, or immediately on discontinuing TYR sphere if the patient stops the trial early. An evaluation of the patient's previous protein substitute and usual GI symptoms are recorded on day one, before the first intake of TYR sphere.
Metabolic control, measured via dried blood spots taken at both visits, and weekly during the 28 days study period, is another primary outcome measure alongside acceptability.
The trial's secondary endpoint is evaluations of growth. Height and weight will be recorded at the Baseline and End of Study visits.
If it is deemed appropriate by the investigator, patients may continue to take the product at the end of the Evaluation Period as part of Care After Research. This continued supply would be given free of charge by the sponsor up until the product is available via a regular prescription, having completed the registration process with the ACBS.
- Conditions
- Tyrosinemias, Alkaptonuria, Tyrosinemia, Type I, Tyrosinemia, Type II, Tyrosinemia, Type III
- Being tested
- TYR sphere
- Keywords
- Tyrosinaemia, AKU, Food for Special Medical Purposes
- Medical subject headings
- Tyrosinemias, Alkaptonuria
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TYR sphereDietary supplement
TYR sphere is a powdered protein substitute for patients with tyrosinaemia.
| Study group | Role | What they receive |
|---|---|---|
| Single Arm | Experimental | All patients will receive TYR sphere, a Food for Special Medical Purposes, as part of thier restricted diet for 28 consecutive days. Receives: Dietary Supplement: TYR sphere |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adherence to prescribed intake of TYR sphere
Patient reported data on actual compared to prescribed intakes of TYR sphere
Measured: Days 1 to 28
Patient record of gastrointestinal tolerance, week 1
Patient reported data on any gastrointestinal symptoms experienced
Measured: Days 1 - 7
Patient record of gastrointestinal tolerance, week 4
Patient reported data on any gastrointestinal symptoms experienced
Measured: Days 21 - 28
Patient assessment of palatability of TYR sphere
Patient reported data on the taste, texture, appearance, smell and ease of use of TYR sphere at the end of the trial period.
Measured: Day 28
Change in metabolic control (tyrosine) over study period
Tyrosine levels obtained from dried blood spots
Measured: One at Baseline Visit (Day 1) one per week during Evaluation Period (Days 1 - 28) and one at End of Study visit (Day 28)
Change in metabolic control (phenylalanine) over study period
Phenylalanine levels obtained from dried blood spots
Measured: One at Baseline Visit (Day 1) one per week during Evaluation Period (Days 1 - 28) and one at End of Study visit (Day 28)
Secondary outcomes (2)
Change in height
Change in height over study period
Measured: Baseline Visit (Day 1) and End of Study Visit (Day 28)
Change in weight
Change in weight over study period
Measured: Baseline Visit (Day 1) and End of Study Visit (Day 28)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Dec 2019
- Study start1 Oct 2019
- Primary completion1 Aug 2020
- Study completion1 Aug 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for TyrosinemiasStudies 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
- Model description
- Single group, open label.
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vitaflo International, Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Anita MacDonald, Principal investigator, Anita.MacDonald@NHS.Net
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04196959
- Sponsor's protocol ID
- MCT-TYRS-2019-03-04
- Status last verified
- Feb 2024
- First submitted
- 6 Dec 2019
- 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
- An Acceptability Study to Evaluate the Adherence, Tolerance and Metabolic Control of Patients With Tyrosinaemia, When Using TYR Sphere (a Food for Special Medical Purposes (FSMP)) as Part of Dietary Management.
- ClinicalTrials.gov ID
- NCT04196959
- Acronym / sponsor ID
- MCT-TYRS-2019-03-04
- Conditions
- Tyrosinemias, Alkaptonuria, Tyrosinemia, Type I, Tyrosinemia, Type II, Tyrosinemia, Type III
- Keywords
- Tyrosinaemia, AKU, Food for Special Medical Purposes
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Vitaflo International, LtdIndustry (pharma, biotech, devices)
- Study start
- 1 Oct 2019 (actual)
- Primary completion
- 1 Aug 2020 (actual)
- Study completion
- 1 Aug 2020 (actual)
- First posted
- 12 Dec 2019 (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.
For 28 days, 20 patients with tyrosinaemia will take TYR sphere as part of the usual dietary management of their condition. Patients will record their adherence to recommended intakes each day, gastrointestinal symptoms (if any) experienced each day of weeks one and four, and the palatability of the product at the end of the trial. Metabolic control will be evaluated by dried blood spots collected during the Baseline and End of Study visits, and weekly during the 28 days of the study itself.
Trial data will be used in an application to make TYR sphere available via the NHS.
Detailed description
TYR sphere is a glycomacropeptide (GMP) based protein substitute for patients with tyrosinaemia.
The Evaluation of TYR sphere study has been designed to generate data required by the United Kingdom's Advisory Committee on Borderline Substances (ACBS), the national regulator for Foods for Special Medical Purposes (FSMP).
The ACBS specify that before a FSMP can be routinely prescribed by the NHS, its acceptability must first be evaluated by a clinical trial in patients for whom it is ultimately designed. The ACBS define acceptability as comprising patient evaluations of the product's palatability, data on their adherence to recommended intakes and their gastrointestinal tolerance of it. TYR sphere is designed for patients of three years of age and upwards. This has informed the trial's age-related inclusion criteria.
Using a mobile phone application, or paper diary form, patients will record their intake of TYR sphere along with the recommended amount on each of the 28 study days. Gastrointestinal tolerance will be recorded each day during weeks one and four, while palatability of TYR sphere will be recorded at the end of the 28 days, or immediately on discontinuing TYR sphere if the patient stops the trial early. An evaluation of the patient's previous protein substitute and usual GI symptoms are recorded on day one, before the first intake of TYR sphere.
Metabolic control, measured via dried blood spots taken at both visits, and weekly during the 28 days study period, is another primary outcome measure alongside acceptability.
The trial's secondary endpoint is evaluations of growth. Height and weight will be recorded at the Baseline and End of Study visits.
If it is deemed appropriate by the investigator, patients may continue to take the product at the end of the Evaluation Period as part of Care After Research. This continued supply would be given free of charge by the sponsor up until the product is available via a regular prescription, having completed the registration process with the ACBS.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single group, open label.
| Arm | Type | Receives |
|---|---|---|
| Single Arm | Experimental | Dietary Supplement: TYR sphereAll patients will receive TYR sphere, a Food for Special Medical Purposes, as part of thier restricted diet for 28 consecutive days. |
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Diagnosed with tyrosinaemia.
- Aged ≥ 3 years.
- In the opinion of the Investigator, can comply with the study protocol and take at least one sachet of the study product per day.
- Willingly given, written, informed consent from patient or parent/guardian.
- Willingly given, written assent (if appropriate).
Key exclusion criteria (6 listed)
- Individuals who are allergic to milk, fish and soya (these allergens are inherent in the study product ingredients).
- Use of additional macro/micronutrient supplements during the study period, unless clinically indicated and prescribed by the Investigator (must be recorded in patient case report form CRF).
- Women who are pregnant / breastfeeding at the start of the study or planning to become pregnant during the study period.
- Individuals who, in the opinion of the investigator, are unable to comply with the requirements of the protocol.
- Any co-morbidity, which, in the opinion of the Investigator, would preclude participation in the study.
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 |
|---|---|---|
| TYR sphereTYR sphere is a powdered protein substitute for patients with tyrosinaemia. | Dietary supplement | Single Arm |
Primary outcome
Adherence to prescribed intake of TYR sphere
Time frame: Days 1 to 28
Patient reported data on actual compared to prescribed intakes of TYR sphere
Patient record of gastrointestinal tolerance, week 1
Time frame: Days 1 - 7
Patient reported data on any gastrointestinal symptoms experienced
Patient record of gastrointestinal tolerance, week 4
Time frame: Days 21 - 28
Patient reported data on any gastrointestinal symptoms experienced
Patient assessment of palatability of TYR sphere
Time frame: Day 28
Patient reported data on the taste, texture, appearance, smell and ease of use of TYR sphere at the end of the trial period.
Change in metabolic control (tyrosine) over study period
Time frame: One at Baseline Visit (Day 1) one per week during Evaluation Period (Days 1 - 28) and one at End of Study visit (Day 28)
Tyrosine levels obtained from dried blood spots
Change in metabolic control (phenylalanine) over study period
Time frame: One at Baseline Visit (Day 1) one per week during Evaluation Period (Days 1 - 28) and one at End of Study visit (Day 28)
Phenylalanine levels obtained from dried blood spots
2 secondary outcomes
Change in height
Time frame: Baseline Visit (Day 1) and End of Study Visit (Day 28)
Change in weight
Time frame: Baseline Visit (Day 1) and End of Study Visit (Day 28)
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
Related trials for Tyrosinemias
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome
Recruiting
GMP Powdered Substitutes in PKU and TYR
Phenylketonuria, Tyrosinemia
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
Anita.MacDonald@NHS.Net
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing TYR sphere in people with Tyrosinemias and Alkaptonuria, run by Vitaflo International, Ltd at 2 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 6 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 2 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?
Adherence to prescribed intake of TYR sphere (time frame: Days 1 to 28). The study lists 6 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.