Experience of Using Lipano in Children With Fatty Acid Oxidation Disorders

Fatty Acid Oxidation Disorder Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04400422
• Other study ID #: Lipano 1 - Case
• Status: Completed
• Phase: N/A
• Start date: June 20, 2018
• Est. completion date: September 20, 2018

Study information

• Verified date: May 2020
• Source: Dr. Schär AG / SPA
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 5
• Est. completion date: September 20, 2018
• Est. primary completion date: September 20, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 18 Months

Eligibility

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

Related Conditions & MeSH terms

• Disease
• Fatty Acid Oxidation Disorder

Intervention

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

Locations

Country	Name	City	State
Russian Federation	Russian Children's Hospital Center for Orphan Diseases	Moscow

Sponsors (1)

Lead Sponsor	Collaborator
Dr. Schär AG / SPA

Country where clinical trial is conducted

Russian Federation, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	Product compliance	Daily questionnaire on amounts offered and amounts actually consumed, compared to recommended amount.	90 days
Primary	Product palatability	Questionnaire data captured to evaluate taste	90 days
Primary	Product acceptability	Brief tick-box questionnaire on overall liking and acceptability of product	90 days
Secondary	Biochemistry stability - creatine kinase	creatine kinase will be monitored	90 days
Secondary	Biochemistry stability - liver enzymes	liver enzymes will be monitored	90 days
Secondary	Anthropometry 1	weight in g monitored to check growth	90 days
Secondary	Anthropometry 2	height in cm monitored to check growth	90 days