Experience of Using Lipano in Children With Fatty Acid Oxidation Disorders

• Sponsor: Dr. Schär AG / SPA (Industry)
• Overall Status: Completed
• CT.gov ID: NCT04400422
• Collaborator: (none)
• Enrollment: 5
• Location: 1
• Arm: 1
• Actual Duration (Months): 3
• Patients Per Site Per Month: 1.7

Study Details

Study Description

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

Condition or Disease	Intervention/Treatment	Phase
Fatty Acid Oxidation Disorder	Dietary Supplement: Lipano formula	N/A

Study Design

• Study Type: Interventional
• Actual Enrollment: 5 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Basic Science
• Official Title: Experience of Using Lipano in Children With LCHAD (Long Chain Fatty Acid Oxidation Disorder)
• Actual Study Start Date: Jun 20, 2018
• Actual Primary Completion Date: Sep 20, 2018
• Actual Study Completion Date: Sep 20, 2018

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

1. Gastrointestinal tolerance: questionnaire [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

2. Product compliance [90 days]

   Daily questionnaire on amounts offered and amounts actually consumed, compared to recommended amount.

3. Product palatability [90 days]

   Questionnaire data captured to evaluate taste

4. Product acceptability [90 days]

   Brief tick-box questionnaire on overall liking and acceptability of product

Secondary Outcome Measures

1. Biochemistry stability - creatine kinase [90 days]

   creatine kinase will be monitored

2. Biochemistry stability - liver enzymes [90 days]

   liver enzymes will be monitored

3. Anthropometry 1 [90 days]

   weight in g monitored to check growth

4. Anthropometry 2 [90 days]

   height in cm monitored to check growth

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A to 18 Months
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Russian Children's Hospital Center for Orphan Diseases	Moscow		Russian Federation	117513

Sponsors and Collaborators

• Dr. Schär AG / SPA

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Dr. Schär AG / SPA
• ClinicalTrials.gov Identifier: NCT04400422
• Other Study ID Numbers: Lipano 1 - Case
• First Posted: May 22, 2020
• Last Update Posted: May 22, 2020
• Last Verified: May 1, 2020

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Disease

Study Results

No Results Posted as of May 22, 2020