Ignite Creation Date:
2024-05-06 @ 8:12 PM
Last Modification Date:
2024-10-26 @ 3:23 PM
Study NCT ID:
NCT06298292
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-03-07
First Post:
2024-03-01
Brief Title:
AcceptabilityTolerance of Protein Substitutes in Tablet Form for the Dietary Management of Rare Aminoacidopathies
Sponsor:
metaX Institut fuer Diatetik GmbH
Organization:
metaX Institut fuer Diatetik GmbH
Study Overview
Official Title:
Zero Minis - Acceptability and Tolerance Market Research
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-03
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
ZeroMinisMR
Brief 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 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 acids which cant 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 parentscaregivers 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 parentsprimary caregivers and taken by the Lead Dietitian Children will also complete an assentconsent form and will receive an information sheet if considered appropriate for their level of understanding
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 acids which cant 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 parentscaregivers 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 parentsprimary caregivers and taken by the Lead Dietitian Children will also complete an assentconsent form and will receive an information sheet if considered appropriate for their level of understanding
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None