Ignite Creation Date:
2024-10-26 @ 3:39 PM
Last Modification Date:
2024-10-26 @ 3:39 PM
Study NCT ID:
NCT06581991
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-08-13
Brief Title:
Liquid Valine and Isoleucine in Maple Syrup Urine Disease
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Valine 50mgml and Isoleucine 25mgml Oral Solutions for Maple Syrup Urine Disease MSUD - Acceptability and Tolerance Study
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 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 locationpreferred venue for the participant and their parentcaregiver Recruitment of each patient will be by written informed consent which will be completed by the parentsprimary 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 normalroutine 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 fooddrink 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 50mgml and isoleucine oral solution 25mgml 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
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 locationpreferred venue for the participant and their parentcaregiver Recruitment of each patient will be by written informed consent which will be completed by the parentsprimary 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 normalroutine 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 fooddrink 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 50mgml and isoleucine oral solution 25mgml 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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None