Experience of Using Lipano in Children With LCHAD (Long Chain Fatty Acid Oxidation Disorder)
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Sponsor
- Enrollment
- 5
- Locations
- 1
- Primary Endpoint
- Gastrointestinal tolerance: questionnaire
Study Overview
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
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Basic Science
- Masking
- None
Eligibility Criteria
- Ages
- — to 18 Months (Child)
- Sex
- All
- Accepts 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
Arms & Interventions
Lipano MCT formula
Kanso Lipano will be consumed daily for 3 months each to assess tolerability and compliance
Intervention: Lipano formula (Dietary Supplement)
Outcomes
Primary Outcomes
Gastrointestinal tolerance: questionnaire
Time Frame: 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
Time Frame: 90 days
Daily questionnaire on amounts offered and amounts actually consumed, compared to recommended amount.
Product palatability
Time Frame: 90 days
Questionnaire data captured to evaluate taste
Product acceptability
Time Frame: 90 days
Brief tick-box questionnaire on overall liking and acceptability of product
Secondary Outcomes
- Biochemistry stability - creatine kinase(90 days)
- Biochemistry stability - liver enzymes(90 days)
- Anthropometry 1(90 days)
- Anthropometry 2(90 days)