Experience of Using Lipano in Children With Fatty Acid Oxidation Disorders

May 21, 2020 updated by: Dr. Schär AG / SPA

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

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 Overview

Status

Completed

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

5

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

      • Moscow, Russian Federation, 117513
        • Russian Children's Hospital Center for Orphan Diseases

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 1 year (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Basic Science
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Lipano MCT formula
Kanso Lipano will be consumed daily for 3 months each to assess tolerability and compliance
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
90 days
Product compliance
Time Frame: 90 days
Daily questionnaire on amounts offered and amounts actually consumed, compared to recommended amount.
90 days
Product palatability
Time Frame: 90 days
Questionnaire data captured to evaluate taste
90 days
Product acceptability
Time Frame: 90 days
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
Time Frame: 90 days
creatine kinase will be monitored
90 days
Biochemistry stability - liver enzymes
Time Frame: 90 days
liver enzymes will be monitored
90 days
Anthropometry 1
Time Frame: 90 days
weight in g monitored to check growth
90 days
Anthropometry 2
Time Frame: 90 days
height in cm monitored to check growth
90 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 20, 2018

Primary Completion (Actual)

September 20, 2018

Study Completion (Actual)

September 20, 2018

Study Registration Dates

First Submitted

May 18, 2020

First Submitted That Met QC Criteria

May 21, 2020

First Posted (Actual)

May 22, 2020

Study Record Updates

Last Update Posted (Actual)

May 22, 2020

Last Update Submitted That Met QC Criteria

May 21, 2020

Last Verified

May 1, 2020

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Lipano 1 - Case

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

IPD Plan Description

This study will be used to demonstrate acceptability for registration and reimbursement of these foods for special medical purposes.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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