Experience of Using Lipano in Children With Fatty Acid Oxidation Disorders

Overview

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

Full Title of Study: “Experience of Using Lipano in Children With LCHAD (Long Chain Fatty Acid Oxidation Disorder)”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Basic Science
    • Masking: None (Open Label)
  • Study Primary Completion Date: September 20, 2018

Interventions

  • Dietary Supplement: Lipano 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

Arms, Groups and Cohorts

  • Experimental: Lipano MCT formula
    • Kanso Lipano will be consumed daily for 3 months each to assess tolerability and compliance

Clinical Trial Outcome Measures

Primary Measures

  • 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 Measures

  • Biochemistry stability – creatine kinase
    • Time Frame: 90 days
    • creatine kinase will be monitored
  • Biochemistry stability – liver enzymes
    • Time Frame: 90 days
    • liver enzymes will be monitored
  • Anthropometry 1
    • Time Frame: 90 days
    • weight in g monitored to check growth
  • Anthropometry 2
    • Time Frame: 90 days
    • height in cm monitored to check growth

Participating in This Clinical Trial

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

Gender Eligibility: All

Minimum Age: N/A

Maximum Age: 18 Months

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Dr. Schär AG / SPA
  • Provider of Information About this Clinical Study
    • Sponsor

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