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Acceptability/Tolerance of Protein Substitutes in Tablet Form for the Dietary Management of Rare Aminoacidopathies
Sponsor: metaX Institut fuer Diatetik GmbH
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.
Official title: Zero Minis - Acceptability and Tolerance Market Research
Key Details
Gender
All
Age Range
7 Years - 18 Years
Study Type
OBSERVATIONAL
Enrollment
15
Start Date
2024-04-01
Completion Date
2026-06-30
Last Updated
2024-03-07
Healthy Volunteers
No
Conditions
Interventions
Zero minis (range of protein substitutes in tablet form)
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.