Tyrosinemia, Type IZeroMinisMRNCT06298292
Acceptability/Tolerance of Protein Substitutes in Tablet Form for the Dietary Management of Rare Aminoacidopathies
An observational study of Tyrosinemia, Type I and Tyrosinemia, Type II, run by metaX Institut fuer Diatetik GmbH.
Trial summary
The purpose of this prospective, observational study is to evaluate the tolerability and acceptability of Zero minis, a range of protein substitute tablets for use in the dietary management of children with either TYROSINAEMIA Type I, II, III or ALKAPTONURIA, HOMOCYSTINURIA, or MAPLE SYRUP URINE DISEASE (MSUD) over… Read more about this study
- Who is it for?
- Ages 7 to 18 years
- Conditions the study is for
- Tyrosinemia, Type I, Tyrosinemia, Type II, Tyrosinemia, Type III
- What is being tested?
- Zero minis (range of protein substitutes in tablet form)
- Type of treatment
- Pill or oral medicine
- How many people
- 15 participants
- Where
- No sites listed yet
- Study dates
- 1 Apr 2024to 30 Jun 2026
- 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: 15 children out of a patient pool with proven tyrosinaemia type I, II, III or alkaptonuria, homocystinuria, or MSUD will be recruited from two specialist metabolic centres.
Who may be able to join
- Diagnosis of Tyrosinaemia type I, II III or Alkaptonuria requiring a tyrosine- and phenylalanine-free protein substitute.
- Diagnosis of Homocystinuria requiring a methionine-free, cystine-enriched protein substitute.
- Diagnosis of MSUD requiring a valine-, leucine- and isoleucine-free protein substitute.
- Subjects who are already taking a protein substitute for one of the specified rare metabolic disorders 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
No sites are listed yet.
About this study
Official title: Zero Minis - Acceptability and Tolerance Market Research
The purpose of this prospective, observational study is to evaluate the tolerability and acceptability of Zero minis, a range of protein substitute tablets for use in the dietary management of children with either TYROSINAEMIA Type I, II, III or ALKAPTONURIA, HOMOCYSTINURIA, or MAPLE SYRUP URINE DISEASE (MSUD) over the age of 7 years.
Read the detailed description
The principle treatment for children with inborn amino acid disorders is a strict low protein diet. Part of this treatment requires the administration of a protein substitute in order to meet basic protein requirements for normal growth and development. Several brands of protein substitutes for inborn amino acid disorders are already available in various presentations. However, compliance with taking protein substitutes continues to be a challenge. As a low protein diet is usually recommended for life, long term compliance is always a major concern. As a result, improving the choice in terms of product type may aid compliance.
Zero minis are a range of protein substitute tablets specifically designed for use in the dietary management of rare amino acid disorders like Tyrosinaemia type I, II, III or Alkaptonuria, Homocystinuria, and MSUD in children from ≥3 years of age onwards. Zero minis are concentrated protein substitutes containing a mixture of pure amino acids (except the amino acid(s) which can´t be metabolized) enriched with vitamins, minerals and trace elements. It is anticipated that they will be a suitable alternative choice for patients with rare metabolic disorders including: tyrosinaemia type I, II, III or alkaptonuria, homocystinuria, or MSUD, broadening the variety of protein substitutes they can choose from in order to suit their lifestyle and preferences.
This is a prospective, multicentre, observational tolerance study with 15 children with rare metabolic disorders. Subjects who are currently taking a protein substitute for the dietary management of either tyrosinaemia type I, II, III or alkaptonuria, homocystinuria, or MSUD, will be recruited for a 7-day trial taking the new ready-to-use protein substitute tablets to evaluate the tolerability and acceptability of the study products. Subjects will replace some or all of their usual protein substitute with the new product suitable for their diagnosed rare metabolic disease.
During the 7-day 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, presentation and packaging of each product; 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 blood tests as is routine in rare metabolic disorders. The results whilst on the study product will be compared with results whilst on their usual protein substitute.
15 children out of a patient pool with tyrosinaemia type I, II, III or alkaptonuria, homocystinuria, or MSUD 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.
- Conditions
- Tyrosinemia, Type I, Tyrosinemia, Type II, Tyrosinemia, Type III, Alkaptonuria, Homocystine; Metabolic Disorder, MSUD (Maple Syrup Urine Disease)
- Being tested
- Zero minis (range of protein substitutes in tablet form)
- Medical subject headings
- Tyrosinemias, Alkaptonuria, Metabolic Diseases, Ketosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Zero minis (range of protein substitutes in tablet form)Dietary supplement
Also called ZeroTP minis, ZeroMet minis, ZeroVIL minis
Intervention: range of protein substitute tablets. Subjects who currently take a protein substitute for the dietary management of either tyrosinaemias or alkaptonuria, homocystinuria, or MSUD will be recruited. Subjects will take the study product for 7 days. Daily questionnaires will be completed (ease of preparation,administration; any problems or gastrointestinal effects). Subjects will replace some or their entire usual protein substitute with the new product suitable for their diagnosed rare metabolic disease. The amount of tablets prescribed will be calculated to provide the same amount of protein as their usual protein substitute. Additional questions at the beginning and end of the study will record information on organoleptic properties, presentation and packaging of the product. Routine weekly blood samples will be collected and analysed for amino acids typically for the metabolic control of the individual specified metabolic disorders as is usual clinical practice.
| Study group | Role | What they receive |
|---|---|---|
| ZeroTP minis | Intervention: Tyrosine-free and Phenylalanine-free protein substitute in tablet form. Subjects who currently take a protein substitute for the dietary management of either tyrosinaemia I, II, III or alkaptonuria will be recruited. Subjects will take the study product for 7 days. Subjects will replace some or their entire usual protein substitute with the new product. The amount of tablets prescribed will be calculated to provide the same amount of protein as their usual protein substitute. Receives: Dietary Supplement: Zero minis (range of protein substitutes in tablet form) | |
| ZeroMet minis | Intervention: Methionine-free and Cystine-enriched protein substitute in tablet form. Subjects who currently take a protein substitute for the dietary management of homocystinuria will be recruited. Subjects will take the study product for 7 days. Subjects will replace some or their entire usual protein substitute with the new product. The amount of tablets prescribed will be calculated to provide the same amount of protein as their usual protein substitute. Receives: Dietary Supplement: Zero minis (range of protein substitutes in tablet form) | |
| ZeroVIL minis | Intervention: Valine-, Isoleucine- and Leucine-free protein substitute in tablet form. Subjects who currently take a protein substitute for the dietary management of MSUD will be recruited. Subjects will take the study product for 7 days. Subjects will replace some or their entire usual protein substitute with the new product. The amount of tablets prescribed will be calculated to provide the same amount of protein as their usual protein substitute. Receives: Dietary Supplement: Zero minis (range of protein substitutes in tablet form) |
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 the amount of consumed study product vs the prescribed doses.
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 blood samples will be collected and analysed for specific amino acids typically for the metabolic control of the individual specified metabolic disorders as is usual clinical practice.
Measured: 7 days
Incidence of study product emergent events.
All adverse events will be recorded throughout the study (as and when they occur). There is no specific time frame as the frequency of adverse events can not be predicted. But as the study product is already commercially available occurence of any adverse event is unlikely.
Measured: 7 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2024
- Study start1 Apr 2024 (estimated)
- Primary completion31 Mar 2026 (estimated)
- Study completion30 Jun 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Tyrosinemia, Type IStudies 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-control
- Time perspective
- Prospective
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- metaX Institut fuer Diatetik GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Birmingham Women's and Children's NHS Foundation Trust, Great Ormond Street Hospital for Children NHS Foundation Trust
- Responsible party
- Sponsor
- Investigators
- Anita MacDonald, Professor, Principal investigator, Birmingham Children´s Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06298292
- Sponsor's protocol ID
- ZerominisCT01
- Acronym
- ZeroMinisMR
- Status last verified
- Mar 2024
- First submitted
- 1 Mar 2024
- Last updated
- 7 Mar 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: No IPD will be shared with other researchers.
Study at a glance
From the registry record, last updated by the sponsor on 7 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Zero Minis - Acceptability and Tolerance Market Research
- ClinicalTrials.gov ID
- NCT06298292
- Acronym / sponsor ID
- ZeroMinisMR / ZerominisCT01
- Conditions
- Tyrosinemia, Type I, Tyrosinemia, Type II, Tyrosinemia, Type III, Alkaptonuria, Homocystine; Metabolic Disorder, MSUD (Maple Syrup Urine Disease)
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- metaX Institut fuer Diatetik GmbHIndustry (pharma, biotech, devices)
- Collaborators
- Birmingham Women's and Children's NHS Foundation Trust, Great Ormond Street Hospital for Children NHS Foundation Trust
- Study start
- 1 Apr 2024 (estimated)
- Primary completion
- 31 Mar 2026 (estimated)
- Study completion
- 30 Jun 2026 (estimated)
- First posted
- 7 Mar 2024 (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 Zero minis, a range of protein substitute tablets for use in the dietary management of children with either TYROSINAEMIA Type I, II, III or ALKAPTONURIA, HOMOCYSTINURIA, or MAPLE SYRUP URINE DISEASE (MSUD) over the age of 7 years.
Detailed description
The principle treatment for children with inborn amino acid disorders is a strict low protein diet. Part of this treatment requires the administration of a protein substitute in order to meet basic protein requirements for normal growth and development. Several brands of protein substitutes for inborn amino acid disorders are already available in various presentations. However, compliance with taking protein substitutes continues to be a challenge. As a low protein diet is usually recommended for life, long term compliance is always a major concern. As a result, improving the choice in terms of product type may aid compliance.
Zero minis are a range of protein substitute tablets specifically designed for use in the dietary management of rare amino acid disorders like Tyrosinaemia type I, II, III or Alkaptonuria, Homocystinuria, and MSUD in children from ≥3 years of age onwards. Zero minis are concentrated protein substitutes containing a mixture of pure amino acids (except the amino acid(s) which can´t be metabolized) enriched with vitamins, minerals and trace elements. It is anticipated that they will be a suitable alternative choice for patients with rare metabolic disorders including: tyrosinaemia type I, II, III or alkaptonuria, homocystinuria, or MSUD, broadening the variety of protein substitutes they can choose from in order to suit their lifestyle and preferences.
This is a prospective, multicentre, observational tolerance study with 15 children with rare metabolic disorders. Subjects who are currently taking a protein substitute for the dietary management of either tyrosinaemia type I, II, III or alkaptonuria, homocystinuria, or MSUD, will be recruited for a 7-day trial taking the new ready-to-use protein substitute tablets to evaluate the tolerability and acceptability of the study products. Subjects will replace some or all of their usual protein substitute with the new product suitable for their diagnosed rare metabolic disease.
During the 7-day 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, presentation and packaging of each product; 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 blood tests as is routine in rare metabolic disorders. The results whilst on the study product will be compared with results whilst on their usual protein substitute.
15 children out of a patient pool with tyrosinaemia type I, II, III or alkaptonuria, homocystinuria, or MSUD 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-control
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| ZeroTP minis | – | Dietary Supplement: Zero minis (range of protein substitutes in tablet form)Intervention: Tyrosine-free and Phenylalanine-free protein substitute in tablet form. Subjects who currently take a protein substitute for the dietary management of either tyrosinaemia I, II, III or alkaptonuria will be recruited. Subjects will take the study product for 7 days. Subjects will replace some or their entire usual protein substitute with the new product. The amount of tablets prescribed will be calculated to provide the same amount of protein as their usual protein substitute. |
| ZeroMet minis | – | Dietary Supplement: Zero minis (range of protein substitutes in tablet form)Intervention: Methionine-free and Cystine-enriched protein substitute in tablet form. Subjects who currently take a protein substitute for the dietary management of homocystinuria will be recruited. Subjects will take the study product for 7 days. Subjects will replace some or their entire usual protein substitute with the new product. The amount of tablets prescribed will be calculated to provide the same amount of protein as their usual protein substitute. |
| ZeroVIL minis | – | Dietary Supplement: Zero minis (range of protein substitutes in tablet form)Intervention: Valine-, Isoleucine- and Leucine-free protein substitute in tablet form. Subjects who currently take a protein substitute for the dietary management of MSUD will be recruited. Subjects will take the study product for 7 days. Subjects will replace some or their entire usual protein substitute with the new product. The amount of tablets prescribed will be calculated to provide the same amount of protein as their usual protein substitute. |
Who may qualify
- Ages
- 7 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- 15 children out of a patient pool with proven tyrosinaemia type I, II, III or alkaptonuria, homocystinuria, or MSUD will be recruited from two specialist metabolic centres.
Key inclusion criteria (6 listed)
- Diagnosis of Tyrosinaemia type I, II III or Alkaptonuria requiring a tyrosine- and phenylalanine-free protein substitute.
- Diagnosis of Homocystinuria requiring a methionine-free, cystine-enriched protein substitute.
- Diagnosis of MSUD requiring a valine-, leucine- and isoleucine-free protein substitute.
- Subjects who are already taking a protein substitute for one of the specified rare metabolic disorders and are willing to try the study product for 7 days.
- Children aged 7 years and over.
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 |
|---|---|---|
| Zero minis (range of protein substitutes in tablet form)Also: ZeroTP minis, ZeroMet minis, ZeroVIL minisIntervention: range of protein substitute tablets. Subjects who currently take a protein substitute for the dietary management of either tyrosinaemias or alkaptonuria, homocystinuria, or MSUD will be recruited. Subjects will take the study product for 7 days. Daily questionnaires will be completed (ease of preparation,administration; any problems or gastrointestinal effects). Subjects will replace some or their entire usual protein substitute with the new product suitable for their diagnosed rare metabolic disease. The amount of tablets prescribed will be calculated to provide the same amount of protein as their usual protein substitute. Additional questions at the beginning and end of the study will record information on organoleptic properties, presentation and packaging of the product. Routine weekly blood samples will be collected and analysed for amino acids typically for the metabolic control of the individual specified metabolic disorders as is usual clinical practice. | Dietary supplement | ZeroMet minis, ZeroTP minis, ZeroVIL minis |
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 the amount of consumed study product vs the prescribed doses.
4 secondary outcomes
Treatment-Emergent tolerability
Time frame: 7 days
Patient Acceptability
Time frame: 7 days
Metabolic control
Time frame: 7 days
Incidence of study product emergent events.
Time frame: 7 days
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Mar 2024.
- Individual participant dataSharing planned: No. No IPD will be shared with other researchers.
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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, Professor principal investigator
Birmingham Children´s Hospital
Questions about this trial
What is this clinical trial studying?
An observational study of Tyrosinemia, Type I and Tyrosinemia, Type II, run by metaX Institut fuer Diatetik GmbH.
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 6 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: status not confirmed, as of the sponsor's last update on 7 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Bernhard Hoffmann, PhD (+49 6031 166 72 71, behoffmann@metax.org), or a site near the patient. 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 7 Mar 2024.