Experience of Using Lipano in Children With Fatty Acid Oxidation Disorders
1 other identifier
interventional
5
1 country
1
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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for not_applicable
Started Jun 2018
Shorter than P25 for not_applicable
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
June 20, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 20, 2018
CompletedStudy Completion
Last participant's last visit for all outcomes
September 20, 2018
CompletedFirst Submitted
Initial submission to the registry
May 18, 2020
CompletedFirst Posted
Study publicly available on registry
May 22, 2020
CompletedMay 22, 2020
May 1, 2020
3 months
May 18, 2020
May 21, 2020
Conditions
Outcome Measures
Primary Outcomes (4)
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 Outcomes (4)
Biochemistry stability - creatine kinase
90 days
Biochemistry stability - liver enzymes
90 days
Anthropometry 1
90 days
Anthropometry 2
90 days
Study Arms (1)
Lipano MCT formula
EXPERIMENTALKanso Lipano will be consumed daily for 3 months each to assess tolerability and compliance
Interventions
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
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Russian Children's Hospital Center for Orphan Diseases
Moscow, 117513, Russia
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- NA
- Masking
- NONE
- Purpose
- BASIC SCIENCE
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 18, 2020
First Posted
May 22, 2020
Study Start
June 20, 2018
Primary Completion
September 20, 2018
Study Completion
September 20, 2018
Last Updated
May 22, 2020
Record last verified: 2020-05
Data Sharing
- IPD Sharing
- Will not share
This study will be used to demonstrate acceptability for registration and reimbursement of these foods for special medical purposes.