This is a market research, observational study to evaluate the tolerability and acceptability of an MCT based formula for infants and young people with long chain fatty acid oxidation disorder from birth to 18 months. Patients with a confirmed diagnosis of a fatty acid oxidation disorder, which requires a specialist diet including MCT will be included in this study
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
BASIC_SCIENCE
Masking
NONE
Enrollment
5
Subjects will be asked to take an MCT infant formula to assess the tolerability, compliance and overall acceptability of the product for patient with LCHAD
Russian Children's Hospital Center for Orphan Diseases
Moscow, Russia
Gastrointestinal tolerance: questionnaire
Daily questionnaire detailing any GI symptoms, severity and change from usual The collection of daily data about the gastrointestinal tolerance of the product
Time frame: 90 days
Product compliance
Daily questionnaire on amounts offered and amounts actually consumed, compared to recommended amount.
Time frame: 90 days
Product palatability
Questionnaire data captured to evaluate taste
Time frame: 90 days
Product acceptability
Brief tick-box questionnaire on overall liking and acceptability of product
Time frame: 90 days
Biochemistry stability - creatine kinase
creatine kinase will be monitored
Time frame: 90 days
Biochemistry stability - liver enzymes
liver enzymes will be monitored
Time frame: 90 days
Anthropometry 1
weight in g monitored to check growth
Time frame: 90 days
Anthropometry 2
height in cm monitored to check growth
Time frame: 90 days
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