TyrosinemiaTYR sphere FraNCT04761588
Evaluation of TYR Sphere in France
A study testing Tyr sphere in people with Tyrosinemia and AKU, run by Vitaflo International, Ltd at 1 site in France.
Trial summary
10 participants aged 3 years and over with tyrosinaemia or alkaptonuria will be prescribed Tyr sphere following an assessment of their individual needs by their dietitian. All participants will enter a 4-week evaluation period, assessing adherence and gastrointestinal tolerance. Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Tyrosinemia, AKU
- What is being tested?
- Tyr sphere
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteFrance
- Study dates
- 1 Dec 2021to 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 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 evaluation 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.
- N.B.: Women who become pregnant during the study will no longer be able to participate and will be withdrawn.
- 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
1 site in 1 country.
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) as Part of Dietary Management.
10 participants aged 3 years and over with tyrosinaemia or alkaptonuria will be prescribed Tyr sphere following an assessment of their individual needs by their dietitian.
All participants will enter a 4-week evaluation period, assessing adherence and gastrointestinal tolerance. Evaluations of Tyr sphere's palatability are made at the end of the evaluation period. Dried blood spots are taken on days 1 and 28 and once per week in between.
Participants who continue to take the product at the end of their evaluation period will enter a follow-up period during which metabolic control, anthropometric and nutritional status data will be collected during the yearly standard of care routine visits.
Read the detailed description
10 participants with tyrosinaemia or alkaptonuria will be invited to participate in this study. Following consent they will be assessed by their dietitian and a recommended amount of Tyr sphere will be prescribed based on their individual needs. Tyr sphere is a food for special medical purposes as defined by Regulation (EU) No 609/2013. It is designed to be prescribed based on its protein content, not its energy content.
Visit 1, baseline clinic visit activities: consent, demographics, anthropometric measurements, gastrointestinal history, dietary assessment, metabolic control assessment including collection of dried blood spots for phenylalanine and tyrosine levels, record routinely taken urine succinylacetone, serum albumin and pre-albumin levels, new dietary prescription.
Weeks 1 to 4 (patient at home): dried blood spots, adverse events reporting/assessment, daily Tyr sphere adherence diary, daily gastrointestinal symptoms diary (weeks 1 and 4 only), daily intake diary for Nitisinone which is routinely prescribed. There will be a telephone call
Visit 2, end of acceptability phase clinic visit activities: anthropometric measurements, review of diaries, review of adverse events, metabolic control assessment including collection of dried blood spots for phenylalanine and tyrosine levels, investigator's assessment of Tyr sphere for the participant and decision on continuing with the prescription.
Three-year routine follow-up: for the participants that do continue with the Tyr sphere prescription (in consultation with the investigator), they will enter a follow-up period of three years, during which only routine, standard of care visits and procedures will take place. Data will be collected on anthropometrics, phenylalanine and tyrosine levels from dried blood spots, adverse events, record routinely taken urine succinylacetone, serum albumin and pre-albumin levels.
- Conditions
- Tyrosinemia, AKU
- Being tested
- Tyr sphere
- Keywords
- Tyrosinemia, AKU, Alkaptonuria, Tyr Sphere, FSMP, 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, low phenylalanine and tyrosine protein substitute, containing a balanced mix of casein glycomacropeptide (cGMP) isolate, essential and non-essential amino acids, carbohydrate, fat, vitamins, minerals and the long chain polyunsaturated fatty acid (LCP) and docosahexaenoic acid (DHA). It contains sugars and sweetener.
The product is designed to be prescribed based on its protein content, not its energy content.
| Study group | Role | What they receive |
|---|---|---|
| Tyr sphere | Experimental | All patients to receive Tyr sphere as part of their dietary management for tyrosinaemia or alkaptonuria (AKU). Receives: Dietary Supplement: Tyr sphere |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in adherence to recommended amounts of Tyr sphere
Patient reported data on actual compared to prescribed intakes of Tyr sphere, assessed by HCPs periodically as per routine practice.
Measured: Recorded daily on days 1-28
Change in gastrointestinal tolerance
Patient reported data on any gastrointestinal symptoms experienced, assessed by HCPs periodically as per routine practice.
Measured: Recorded daily on days 1-28
Patient evaluation of Tyr sphere's palatability
Patient-reported assessment of Tyr sphere's palatability on a Likert scale: 1 (really didn't like it) to 5 (loved it).
Measured: Day 28, end of acceptability phase
Change in metabolic control: tyrosine levels
Tyrosine levels obtained from dried blood spots
Measured: Day 1, week 1, week 2, week 3, week 4, day 28
Change in metabolic control: phenylalanine levels
Phenylalanine levels obtained from dried blood spots
Measured: Day 1, week 1, week 2, week 3, week 4, day 28
Secondary outcomes (4)
Long term change in growth
Recorded progress in growth from standard of care follow-up visits for three years
Measured: 3-year follow-up period
Long term change in urine succinylacetone level
Recorded changes in urine succinylacetone levels from standard of care follow-up visits for three years
Measured: 3-year follow-up period
Long term change in pre-albumin level
Recorded changes in pre-albumin levels from standard of care follow-up visits for three years
Measured: 3-year follow-up period
Long term change in serum albumin level
Recorded changes in serum albumin levels from standard of care follow-up visits for three years
Measured: 3-year 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 posted21 Feb 2021
- Study start1 Dec 2021
- Primary completion31 Dec 2024
- 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 TyrosinemiaStudies 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
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vitaflo International, Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jean-Baptiste Arnoux, Principal investigator, Hopital Necker-Enfants Malades
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04761588
- Sponsor's protocol ID
- TyrSph-2020-10-16
- Other IDs
- SI 21.01.06.74121, 2021-A01263-38
- Acronym
- TYR sphere Fra
- Status last verified
- Nov 2025
- First submitted
- 15 Feb 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 Acceptability Study to Evaluate the Adherence, Tolerance and Metabolic Control of Patients With Tyrosinaemia When Using TYR Sphere (a Food for Special Medical Purposes) as Part of Dietary Management.
- ClinicalTrials.gov ID
- NCT04761588
- Acronym / sponsor ID
- TYR sphere Fra / TyrSph-2020-10-16
- Conditions
- Tyrosinemia, AKU
- Keywords
- Tyrosinemia, AKU, Alkaptonuria, Tyr Sphere, FSMP, Food for special medical purposes
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Vitaflo International, LtdIndustry (pharma, biotech, devices)
- Study start
- 1 Dec 2021 (actual)
- Primary completion
- 31 Dec 2024 (actual)
- Study completion
- 31 Oct 2025 (actual)
- First posted
- 21 Feb 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.
10 participants aged 3 years and over with tyrosinaemia or alkaptonuria will be prescribed Tyr sphere following an assessment of their individual needs by their dietitian.
All participants will enter a 4-week evaluation period, assessing adherence and gastrointestinal tolerance. Evaluations of Tyr sphere's palatability are made at the end of the evaluation period. Dried blood spots are taken on days 1 and 28 and once per week in between.
Participants who continue to take the product at the end of their evaluation period will enter a follow-up period during which metabolic control, anthropometric and nutritional status data will be collected during the yearly standard of care routine visits.
Detailed description
10 participants with tyrosinaemia or alkaptonuria will be invited to participate in this study. Following consent they will be assessed by their dietitian and a recommended amount of Tyr sphere will be prescribed based on their individual needs. Tyr sphere is a food for special medical purposes as defined by Regulation (EU) No 609/2013. It is designed to be prescribed based on its protein content, not its energy content.
Visit 1, baseline clinic visit activities: consent, demographics, anthropometric measurements, gastrointestinal history, dietary assessment, metabolic control assessment including collection of dried blood spots for phenylalanine and tyrosine levels, record routinely taken urine succinylacetone, serum albumin and pre-albumin levels, new dietary prescription.
Weeks 1 to 4 (patient at home): dried blood spots, adverse events reporting/assessment, daily Tyr sphere adherence diary, daily gastrointestinal symptoms diary (weeks 1 and 4 only), daily intake diary for Nitisinone which is routinely prescribed. There will be a telephone call
Visit 2, end of acceptability phase clinic visit activities: anthropometric measurements, review of diaries, review of adverse events, metabolic control assessment including collection of dried blood spots for phenylalanine and tyrosine levels, investigator's assessment of Tyr sphere for the participant and decision on continuing with the prescription.
Three-year routine follow-up: for the participants that do continue with the Tyr sphere prescription (in consultation with the investigator), they will enter a follow-up period of three years, during which only routine, standard of care visits and procedures will take place. Data will be collected on anthropometrics, phenylalanine and tyrosine levels from dried blood spots, adverse events, record routinely taken urine succinylacetone, serum albumin and pre-albumin levels.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Tyr sphere | Experimental | Dietary Supplement: Tyr sphereAll patients to receive Tyr sphere as part of their dietary management for tyrosinaemia or alkaptonuria (AKU). |
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 (7 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 evaluation 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.
- N.B.: Women who become pregnant during the study will no longer be able to participate and will be withdrawn.
- Individuals who, in the opinion of the investigator, are unable to comply with the requirements of the protocol.
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, low phenylalanine and tyrosine protein substitute, containing a balanced mix of casein glycomacropeptide (cGMP) isolate, essential and non-essential amino acids, carbohydrate, fat, vitamins, minerals and the long chain polyunsaturated fatty acid (LCP) and docosahexaenoic acid (DHA). It contains sugars and sweetener. The product is designed to be prescribed based on its protein content, not its energy content. | Dietary supplement | Tyr sphere |
Primary outcome
Change in adherence to recommended amounts of Tyr sphere
Time frame: Recorded daily on days 1-28
Patient reported data on actual compared to prescribed intakes of Tyr sphere, assessed by HCPs periodically as per routine practice.
Change in gastrointestinal tolerance
Time frame: Recorded daily on days 1-28
Patient reported data on any gastrointestinal symptoms experienced, assessed by HCPs periodically as per routine practice.
Patient evaluation of Tyr sphere's palatability
Time frame: Day 28, end of acceptability phase
Patient-reported assessment of Tyr sphere's palatability on a Likert scale: 1 (really didn't like it) to 5 (loved it).
Change in metabolic control: tyrosine levels
Time frame: Day 1, week 1, week 2, week 3, week 4, day 28
Tyrosine levels obtained from dried blood spots
Change in metabolic control: phenylalanine levels
Time frame: Day 1, week 1, week 2, week 3, week 4, day 28
Phenylalanine levels obtained from dried blood spots
4 secondary outcomes
Long term change in growth
Time frame: 3-year follow-up period
Long term change in urine succinylacetone level
Time frame: 3-year follow-up period
Long term change in pre-albumin level
Time frame: 3-year follow-up period
Long term change in serum albumin level
Time frame: 3-year 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
Related trials for Tyrosinemia
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.
Jean-Baptiste Arnoux principal investigator
Hopital Necker-Enfants Malades
1 site 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 Tyrosinemia and AKU, 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 5 inclusion and 7 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?
Change in adherence to recommended amounts of Tyr sphere (time frame: Recorded daily on days 1-28). The study lists 5 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.