<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT04400422</url>
  </required_header>
  <id_info>
    <org_study_id>Lipano 1 - Case</org_study_id>
    <nct_id>NCT04400422</nct_id>
  </id_info>
  <brief_title>Experience of Using Lipano in Children With Fatty Acid Oxidation Disorders</brief_title>
  <official_title>Experience of Using Lipano in Children With LCHAD (Long Chain Fatty Acid Oxidation Disorder)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Dr. Schär AG / SPA</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Dr. Schär AG / SPA</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is a market research, observational study to evaluate the tolerability and acceptability&#xD;
      of an MCT based formula for infants and young people with long chain fatty acid oxidation&#xD;
      disorder from birth to 18 months. Patients with a confirmed diagnosis of a fatty acid&#xD;
      oxidation disorder, which requires a specialist diet including MCT will be included in this&#xD;
      study&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">June 20, 2018</start_date>
  <completion_date type="Actual">September 20, 2018</completion_date>
  <primary_completion_date type="Actual">September 20, 2018</primary_completion_date>
  <phase>N/A</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Basic Science</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Gastrointestinal tolerance: questionnaire</measure>
    <time_frame>90 days</time_frame>
    <description>Daily questionnaire detailing any GI symptoms, severity and change from usual The collection of daily data about the gastrointestinal tolerance of the product</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Product compliance</measure>
    <time_frame>90 days</time_frame>
    <description>Daily questionnaire on amounts offered and amounts actually consumed, compared to recommended amount.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Product palatability</measure>
    <time_frame>90 days</time_frame>
    <description>Questionnaire data captured to evaluate taste</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Product acceptability</measure>
    <time_frame>90 days</time_frame>
    <description>Brief tick-box questionnaire on overall liking and acceptability of product</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Biochemistry stability - creatine kinase</measure>
    <time_frame>90 days</time_frame>
    <description>creatine kinase will be monitored</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Biochemistry stability - liver enzymes</measure>
    <time_frame>90 days</time_frame>
    <description>liver enzymes will be monitored</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Anthropometry 1</measure>
    <time_frame>90 days</time_frame>
    <description>weight in g monitored to check growth</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Anthropometry 2</measure>
    <time_frame>90 days</time_frame>
    <description>height in cm monitored to check growth</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">5</enrollment>
  <condition>Fatty Acid Oxidation Disorder</condition>
  <arm_group>
    <arm_group_label>Lipano MCT formula</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Kanso Lipano will be consumed daily for 3 months each to assess tolerability and compliance</description>
  </arm_group>
  <intervention>
    <intervention_type>Dietary Supplement</intervention_type>
    <intervention_name>Lipano formula</intervention_name>
    <description>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</description>
    <arm_group_label>Lipano MCT formula</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Diagnosis of a fatty acid oxidation disorder requiring a ketogenic or a specialist&#xD;
             diet as part of their treatment, which includes MCT.&#xD;
&#xD;
          -  Patients from birth to 18 months&#xD;
&#xD;
          -  Written informed consent obtained from patient or parental caregiver&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Presence of serious concurrent illness&#xD;
&#xD;
          -  Investigator's uncertainty about the willingness or ability of the patient to comply&#xD;
             with the protocol requirements&#xD;
&#xD;
          -  Participation in any other studies involving investigational or marketed products&#xD;
             concomitantly or within two weeks prior to entry into the study.&#xD;
&#xD;
          -  Any patients having taken antibiotics over the previous 2 weeks leading up to the&#xD;
             study&#xD;
&#xD;
          -  Patients over 18 months of age&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>18 Months</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>Russian Children's Hospital Center for Orphan Diseases</name>
      <address>
        <city>Moscow</city>
        <zip>117513</zip>
        <country>Russian Federation</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Russian Federation</country>
  </location_countries>
  <verification_date>May 2020</verification_date>
  <study_first_submitted>May 18, 2020</study_first_submitted>
  <study_first_submitted_qc>May 21, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">May 22, 2020</study_first_posted>
  <last_update_submitted>May 21, 2020</last_update_submitted>
  <last_update_submitted_qc>May 21, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">May 22, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
    <ipd_description>This study will be used to demonstrate acceptability for registration and reimbursement of these foods for special medical purposes.</ipd_description>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT04400422
org study id: Lipano 1 - Case
nct id: NCT04400422
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
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
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Basic Science
masking: None (Open Label)
measure: Gastrointestinal tolerance: questionnaire
time frame: 90 days
description: Daily questionnaire detailing any GI symptoms, severity and change from usual The collection of daily data about the gastrointestinal tolerance of the product
measure: Product compliance
time frame: 90 days
description: Daily questionnaire on amounts offered and amounts actually consumed, compared to recommended amount.
measure: Product palatability
time frame: 90 days
description: Questionnaire data captured to evaluate taste
measure: Product acceptability
time frame: 90 days
description: Brief tick-box questionnaire on overall liking and acceptability of product
measure: Biochemistry stability - creatine kinase
time frame: 90 days
description: creatine kinase will be monitored
measure: Biochemistry stability - liver enzymes
time frame: 90 days
description: liver enzymes will be monitored
measure: Anthropometry 1
time frame: 90 days
description: weight in g monitored to check growth
measure: Anthropometry 2
time frame: 90 days
description: height in cm monitored to check growth
arm group label: Lipano MCT formula
arm group type: Experimental
description: Kanso Lipano will be consumed daily for 3 months each to assess tolerability and compliance
intervention type: Dietary Supplement
intervention name: Lipano formula
description: 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
arm group label: Lipano MCT formula
criteria: 
gender: All
minimum age: N/A
maximum age: 18 Months
healthy volunteers: No
facility: 
country: Russian Federation
responsible party type: Sponsor
sharing ipd: No
ipd description: This study will be used to demonstrate acceptability for registration and reimbursement of these foods for special medical purposes.