- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04400422
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
Conditions
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.
Sponsor
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.
Clinical Trials on Fatty Acid Oxidation Disorder
-
Reneo Pharma LtdCompletedFatty Acid Oxidation DisorderUnited States, Spain, France, Austria, Poland, Belgium, Czechia, Denmark, Hungary, Italy, United Kingdom
-
University of OxfordCompleted
-
Reneo Pharma LtdCompletedFatty Acid Oxidation DisordersUnited States, France, Spain
-
Academisch Medisch Centrum - Universiteit van Amsterdam...Stichting StofwisselkrachtNot yet recruitingLong-chain Fatty Acid Oxidation Disorders \(LC-FAOD\)Netherlands
-
Ultragenyx Pharmaceutical IncRecruitingLong-chain Fatty Acid Oxidation Disorders (LC-FAOD)Turkey, United Kingdom, Spain, Saudi Arabia, Poland, Czechia, Germany
-
Ultragenyx Pharmaceutical IncCompletedLong-chain Fatty Acid Oxidation Disorders (LC-FAOD)United States
-
Ultragenyx Pharmaceutical IncTerminatedLong-chain Fatty Acid Oxidation Disorders (LC-FAOD)United States
-
Ultragenyx Pharmaceutical IncRecruitingLong-chain Fatty Acid Oxidation Disorders (LC-FAOD)United States, Canada
-
University of TorontoCompletedProtein/Amino Acid Metabolism | Indicator Amino Acid Oxidation MethodCanada
-
University of TorontoDairy Management Inc.RecruitingDietary Proteins | Indicator Amino Acid OxidationCanada
Clinical Trials on Lipano formula
-
HiPP GmbH & Co. Vertrieb KGCharite University, Berlin, GermanyCompletedDisturbance of GrowthGermany, Austria, Serbia
-
HiPP GmbH & Co. Vertrieb KGTerminatedInfantile ColicGermany, Italy
-
Dr. Sami Ulus Children's HospitalUnknownGrowth Failure | Neurodevelopmental DisorderTurkey
-
Danone NutriciaNot yet recruiting
-
HiPP GmbH & Co. Vertrieb KGBiofortis Mérieux NutriSciencesTerminated
-
HiPP GmbH & Co. Vertrieb KGActive, not recruitingAtopic DiseasesFrance, Germany, Spain, Czechia, Bulgaria, Finland, Italy, Poland, Portugal, Serbia
-
Boehringer IngelheimCompleted
-
Société des Produits Nestlé (SPN)CompletedFeeding DisorderPhilippines, Hong Kong, Taiwan, Thailand
-
Société des Produits Nestlé (SPN)Completed
-
Abbott NutritionCompleted