Experience of Using Lipano in Children With Fatty Acid Oxidation Disorders

Study Overview
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Brief Summary
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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

Official Title
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Experience of Using Lipano in Children With LCHAD (Long Chain Fatty Acid Oxidation Disorder)

Conditions
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Fatty Acid Oxidation Disorder

Intervention / Treatment
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* Dietary Supplement: Lipano formula


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Maximum Age: 18 Months

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Basic Science
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Lipano MCT formula<br>Kanso Lipano will be consumed daily for 3 months each to assess tolerability and compliance | Dietary Supplement: Lipano formula<br>* 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<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 90 days | 
| Product compliance | Daily questionnaire on amounts offered and amounts actually consumed, compared to recommended amount. | 90 days | 
| Product palatability | Questionnaire data captured to evaluate taste | 90 days | 
| Product acceptability | Brief tick-box questionnaire on overall liking and acceptability of product | 90 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Biochemistry stability - creatine kinase | creatine kinase will be monitored | 90 days | 
| Biochemistry stability - liver enzymes | liver enzymes will be monitored | 90 days | 
| Anthropometry 1 | weight in g monitored to check growth | 90 days | 
| Anthropometry 2 | height in cm monitored to check growth | 90 days |