Fatty Acid Oxidation Disorder
Conditions
Brief summary
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
Interventions
DIETARY_SUPPLEMENTLipano formula
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
Sponsors
Dr. Schär AG / SPA
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
BASIC_SCIENCE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 18 Months
Healthy volunteers
No
Inclusion criteria
* Diagnosis of a fatty acid oxidation disorder requiring a ketogenic or a specialist diet as part of their treatment, which includes MCT. * Patients from birth to 18 months * Written informed consent obtained from patient or parental caregiver
Exclusion criteria
* Presence of serious concurrent illness * Investigator's uncertainty about the willingness or ability of the patient to comply with the protocol requirements * Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study. * Any patients having taken antibiotics over the previous 2 weeks leading up to the study * Patients over 18 months of age
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Product acceptability | 90 days | Brief tick-box questionnaire on overall liking and acceptability of product |
| Gastrointestinal tolerance: questionnaire | 90 days | Daily questionnaire detailing any GI symptoms, severity and change from usual The collection of daily data about the gastrointestinal tolerance of the product |
| Product compliance | 90 days | Daily questionnaire on amounts offered and amounts actually consumed, compared to recommended amount. |
| Product palatability | 90 days | Questionnaire data captured to evaluate taste |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Biochemistry stability - creatine kinase | 90 days | creatine kinase will be monitored |
| Biochemistry stability - liver enzymes | 90 days | liver enzymes will be monitored |
| Anthropometry 1 | 90 days | weight in g monitored to check growth |
| Anthropometry 2 | 90 days | height in cm monitored to check growth |
Countries
Russia
Outcome results
None listed