Maple Syrup Urine DiseaseNCT06581991
Liquid Valine and Isoleucine in Maple Syrup Urine Disease
A study testing Valine oral solution (50mg/ml) and isoleucine oral solution (25mg/ml) in people with Maple Syrup Urine Disease, run by Meta Healthcare Ltd.
Trial summary
This is a prospective, observational research study in 5 children with Maple Syrup Urine Disease (MSUD). Subjects who are currently taking a valine and isoleucine supplement for MSUD will be recruited for a 56 day trial, of a new ready-to-use valine supplement and a new ready-to-use isoleucine supplement, to evaluate… Read more about this study
- Who is it for?
- Ages 12 months to 16 years
- Conditions the study is for
- Maple Syrup Urine Disease
- What is being tested?
- Valine oral solution (50mg/ml) and isoleucine oral solution (25mg/ml)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- No sites listed yet
- Study dates
- Oct 2024to Sep 2026
- 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 12 months to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male and female MSUD patients ≥1 year and <16 years of age.
- Patients diagnosed with MSUD.
- Taking both valine and isoleucine supplements for MSUD and willing to take the new supplements for 56 days.
- Absence of comorbidities.
- Adherence with dietary management and valine and isoleucine supplements.
- Able to understand and comply with the requirements of the investigation and sign the Informed Consent Form/Assent form
Who cannot join
- Age <1 year old and >16 years old.
- Patients with comorbidities.
- Any moderate to severe acute illness which in the opinion of the Investigator would interfere with the study procedures or study outcome.
- History of poor co-operation, non-adherence with dietary management, or poor adherence to investigation procedures.
- Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study.
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Valine 50mg/ml and Isoleucine 25mg/ml Oral Solutions for Maple Syrup Urine Disease (MSUD) - Acceptability and Tolerance Study
This is a prospective, observational research study in 5 children with Maple Syrup Urine Disease (MSUD). Subjects who are currently taking a valine and isoleucine supplement for MSUD will be recruited for a 56 day trial, of a new ready-to-use valine supplement and a new ready-to-use isoleucine supplement, to evaluate the tolerability and acceptability of the study products compared with their usual products.
Read the detailed description
This is a prospective, observational 2-part research study in 5 children with Maple Syrup Urine Disease (MSUD). Subjects who are currently taking a valine and isoleucine supplement for MSUD will be recruited for a 56 day trial, of a new ready-to-use valine supplement (Part 1) and a ready-to-use isoleucine supplement (Part 2), to evaluate the tolerability and acceptability of the study products compared with their usual products.
When an appropriate subject has been identified, they and their parents/ caregivers will be sent a study information sheet via the post. They will be invited to request further information about the study if they wish by contacting the Dietitian. The Dietitian will explain the study in more detail either by telephone or via a face-to-face consultation in a convenient location/preferred venue for the participant and their parent/caregiver. Recruitment of each patient will be by written informed consent, which will be completed by the parents/primary caregivers and taken by the Dietitian. Children will also complete an assent/ consent form. Reimbursement will be offered for any travel expenses incurred for any visits to the hospital that are over and above normal/routine visits.
Subjects will replace their usual daily dose of powdered valine supplement with the equivalent amount of the liquid valine supplement for 28 days (Part 1). They will then return to their usual valine supplement and replace their usual powdered isoleucine supplement with the new liquid isoleucine supplement for a further 28 days (Part 2). The outcome of this assessment will be used in a submission to regulatory authorities to enable the study products to be reimbursable on prescription in the United Kingdom (UK). Subjects will be asked to take the new valine supplement for 28 days (Part 1), then the new isoleucine supplement for 28 days (Part 2). During this time, caregivers will be asked to complete a daily acceptability and tolerance questionnaire recording information on:
- Usage and adherence
- Ease of use and any issues with administration
- Any gastro-intestinal side effects The questions should take a maximum of 10 minutes to complete each day. Additional questions will also be completed at the beginning and end of the study that will consider opinion on both usual valine and isoleucine, and study valine and isoleucine, with respect to taste, appearance, smell, presentation and packaging; ease of administration; how the valine is taken; and any other problems or symptoms.
Weight, height and 3 day dietary intake will also be recorded at the beginning and end of Part 1 and Part 2 of the study. A 3-day food diary will record food/drink intake at the beginning of the study whilst taking their usual supplements (Day -3, -2, -1), for 3 days at the end of Part 1 of the study whilst taking the new valine supplement (Days 26, 27, 28), and for 3 days at the end of Part 2 of the study whilst taking the new isoleucine supplement (Days 54, 55, 56).
Meta healthcare will supply the valine oral solution (50mg/ml) and isoleucine oral solution (25mg/ml) for participants free of charge.
During the 56 day trial, weekly routine blood samples will be posted and analysed for valine, leucine and isoleucine as is usual clinical practice. At recruitment, the previous 4 blood results for valine, leucine and isoleucine will be recorded for comparison.
- Conditions
- Maple Syrup Urine Disease
- Being tested
- Valine oral solution (50mg/ml) and isoleucine oral solution (25mg/ml)
- Keywords
- Maple Syrup Urine Disease, MSUD, Valine, Isoleucine
- Medical subject headings
- Maple Syrup Urine Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Valine oral solution (50mg/ml) and isoleucine oral solution (25mg/ml)Dietary supplement
Subjects will take valine oral solution (50mg/ml) in place of usual powdered valine supplement for 28 days. Subjects will then take isoleucine oral solution (25mg/ml) in place of their usual powdered isoleucine supplement for 28 days.
| Study group | Role | What they receive |
|---|---|---|
| Liquid valine and liquid isoleucine administration | Experimental | Liquid valine given for 28 days, liquid isoleucine for 28 days Receives: Dietary Supplement: Valine oral solution (50mg/ml) and isoleucine oral solution (25mg/ml) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Daily data on gastrointestinal tolerance
A daily record of any gastrointestinal symptoms compared with usual valine and isoleucine supplement
Measured: From enrollment to end of treatment at 56 days
Acceptability and tolerance
Qualitative questionnaires at study start and end of Part 1 (28 days) and Part 2 (56 dys) of the study
Measured: From enrolment to end of treatment at 56
Daily data on study product adherence
A daily record of study product intake (actual versus prescribed) to check for adherence
Measured: From enrollment to the end of treatment at 56 days
3-day food diaries
A record of patient food and fluid intake for 3 days to monitor valine intake from food - at enrollment, end of part 1 and end of part 2
Measured: From enrollment to the end of treatment at 56 days
Blood results for valine, leucine, isoleucine
Routine weekly blood sample taken at home for valine, leucine and isoleucine and posted to the hospital. These will be compared with blood results prior to study commencement to check for any changes
Measured: From enrollment to end of treatment at 56 days
Secondary outcomes (1)
Weight and height measurement
Weight and height will be measured at enrollment, end of Part 1 (28 days) and end of Part 2 (56 days) of the study to check that growth is adequate
Measured: From enrollment to the end of treatment at 56 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Sep 2024
- Study startOct 2024 (estimated)
- Primary completionSep 2026 (estimated)
- Study completionSep 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Maple Syrup Urine DiseaseStudies 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
- Model description
- Prospective, open label, observational study of 5 subjects with MSUD for 56 days
- Masking
- None (open label)
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Meta Healthcare Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Birmingham Women's and Children's NHS Foundation Trust
- Responsible party
- Sponsor
- Investigators
- Anita MacDonald, Principal investigator, Birmingham Children's Hospital, UK
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06581991
- Sponsor's protocol ID
- LVMSUD2022(2)
- Status last verified
- Aug 2024
- First submitted
- 13 Aug 2024
- Last updated
- 3 Sep 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: No identifiable individual patient data will disclosed outside of the clinical care team.
Study at a glance
From the registry record, last updated by the sponsor on 3 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Valine 50mg/ml and Isoleucine 25mg/ml Oral Solutions for Maple Syrup Urine Disease (MSUD) - Acceptability and Tolerance Study
- ClinicalTrials.gov ID
- NCT06581991
- Acronym / sponsor ID
- LVMSUD2022(2)
- Conditions
- Maple Syrup Urine Disease
- Keywords
- Maple Syrup Urine Disease, MSUD, Valine, Isoleucine
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- Meta Healthcare LtdIndustry (pharma, biotech, devices)
- Collaborators
- Birmingham Women's and Children's NHS Foundation Trust
- Study start
- Oct 2024 (estimated)
- Primary completion
- Sep 2026 (estimated)
- Study completion
- Sep 2026 (estimated)
- First posted
- 3 Sep 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.
This is a prospective, observational research study in 5 children with Maple Syrup Urine Disease (MSUD). Subjects who are currently taking a valine and isoleucine supplement for MSUD will be recruited for a 56 day trial, of a new ready-to-use valine supplement and a new ready-to-use isoleucine supplement, to evaluate the tolerability and acceptability of the study products compared with their usual products.
Detailed description
This is a prospective, observational 2-part research study in 5 children with Maple Syrup Urine Disease (MSUD). Subjects who are currently taking a valine and isoleucine supplement for MSUD will be recruited for a 56 day trial, of a new ready-to-use valine supplement (Part 1) and a ready-to-use isoleucine supplement (Part 2), to evaluate the tolerability and acceptability of the study products compared with their usual products.
When an appropriate subject has been identified, they and their parents/ caregivers will be sent a study information sheet via the post. They will be invited to request further information about the study if they wish by contacting the Dietitian. The Dietitian will explain the study in more detail either by telephone or via a face-to-face consultation in a convenient location/preferred venue for the participant and their parent/caregiver. Recruitment of each patient will be by written informed consent, which will be completed by the parents/primary caregivers and taken by the Dietitian. Children will also complete an assent/ consent form. Reimbursement will be offered for any travel expenses incurred for any visits to the hospital that are over and above normal/routine visits.
Subjects will replace their usual daily dose of powdered valine supplement with the equivalent amount of the liquid valine supplement for 28 days (Part 1). They will then return to their usual valine supplement and replace their usual powdered isoleucine supplement with the new liquid isoleucine supplement for a further 28 days (Part 2). The outcome of this assessment will be used in a submission to regulatory authorities to enable the study products to be reimbursable on prescription in the United Kingdom (UK). Subjects will be asked to take the new valine supplement for 28 days (Part 1), then the new isoleucine supplement for 28 days (Part 2). During this time, caregivers will be asked to complete a daily acceptability and tolerance questionnaire recording information on:
- Usage and adherence
- Ease of use and any issues with administration
- Any gastro-intestinal side effects The questions should take a maximum of 10 minutes to complete each day. Additional questions will also be completed at the beginning and end of the study that will consider opinion on both usual valine and isoleucine, and study valine and isoleucine, with respect to taste, appearance, smell, presentation and packaging; ease of administration; how the valine is taken; and any other problems or symptoms.
Weight, height and 3 day dietary intake will also be recorded at the beginning and end of Part 1 and Part 2 of the study. A 3-day food diary will record food/drink intake at the beginning of the study whilst taking their usual supplements (Day -3, -2, -1), for 3 days at the end of Part 1 of the study whilst taking the new valine supplement (Days 26, 27, 28), and for 3 days at the end of Part 2 of the study whilst taking the new isoleucine supplement (Days 54, 55, 56).
Meta healthcare will supply the valine oral solution (50mg/ml) and isoleucine oral solution (25mg/ml) for participants free of charge.
During the 56 day trial, weekly routine blood samples will be posted and analysed for valine, leucine and isoleucine as is usual clinical practice. At recruitment, the previous 4 blood results for valine, leucine and isoleucine will be recorded for comparison.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Prospective, open label, observational study of 5 subjects with MSUD for 56 days
| Arm | Type | Receives |
|---|---|---|
| Liquid valine and liquid isoleucine administration | Experimental | Dietary Supplement: Valine oral solution (50mg/ml) and isoleucine oral solution (25mg/ml)Liquid valine given for 28 days, liquid isoleucine for 28 days |
Who may qualify
- Ages
- 12 months to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Male and female MSUD patients ≥1 year and <16 years of age.
- Patients diagnosed with MSUD.
- Taking both valine and isoleucine supplements for MSUD and willing to take the new supplements for 56 days.
- Absence of comorbidities.
- Adherence with dietary management and valine and isoleucine supplements.
Key exclusion criteria (5 listed)
- Age <1 year old and >16 years old.
- Patients with comorbidities.
- Any moderate to severe acute illness which in the opinion of the Investigator would interfere with the study procedures or study outcome.
- History of poor co-operation, non-adherence with dietary management, or poor adherence to investigation procedures.
- Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into 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 |
|---|---|---|
| Valine oral solution (50mg/ml) and isoleucine oral solution (25mg/ml)Subjects will take valine oral solution (50mg/ml) in place of usual powdered valine supplement for 28 days. Subjects will then take isoleucine oral solution (25mg/ml) in place of their usual powdered isoleucine supplement for 28 days. | Dietary supplement | Liquid valine and liquid isoleucine administration |
Primary outcome
Daily data on gastrointestinal tolerance
Time frame: From enrollment to end of treatment at 56 days
A daily record of any gastrointestinal symptoms compared with usual valine and isoleucine supplement
Acceptability and tolerance
Time frame: From enrolment to end of treatment at 56
Qualitative questionnaires at study start and end of Part 1 (28 days) and Part 2 (56 dys) of the study
Daily data on study product adherence
Time frame: From enrollment to the end of treatment at 56 days
A daily record of study product intake (actual versus prescribed) to check for adherence
3-day food diaries
Time frame: From enrollment to the end of treatment at 56 days
A record of patient food and fluid intake for 3 days to monitor valine intake from food - at enrollment, end of part 1 and end of part 2
Blood results for valine, leucine, isoleucine
Time frame: From enrollment to end of treatment at 56 days
Routine weekly blood sample taken at home for valine, leucine and isoleucine and posted to the hospital. These will be compared with blood results prior to study commencement to check for any changes
1 secondary outcome
Weight and height measurement
Time frame: From enrollment to the end of treatment at 56 days
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Sep 2024.
- Individual participant dataSharing planned: No. No identifiable individual patient data will disclosed outside of the clinical care team.
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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 Children's Hospital, UK
Questions about this trial
What is this clinical trial studying?
A study testing Valine oral solution (50mg/ml) and isoleucine oral solution (25mg/ml) in people with Maple Syrup Urine Disease, run by Meta Healthcare Ltd.
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 3 Sep 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 Anita MacDonald (+44 1213338024, anita.macdonald@nhs.net), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Daily data on gastrointestinal tolerance (time frame: From enrollment to end of treatment at 56 days). 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 3 Sep 2024.