<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/NCT02234024</url>
  </required_header>
  <id_info>
    <org_study_id>DDC-62314</org_study_id>
    <nct_id>NCT02234024</nct_id>
  </id_info>
  <brief_title>Oral Supplementation of Gangliosides to Treat a Rare Metabolic Disorder</brief_title>
  <official_title>Oral Supplementation of Gangliosides - A Potential Treatment for GM3 Synthase Deficiency</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>DDC Clinic - Center for Special Needs Children</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>DDC Clinic - Center for Special Needs Children</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this pilot project is to see if a supplemental form of dietary gangliosides&#xD;
      can serve as a potential treatment for the rare metabolic condition called ganglioside GM3&#xD;
      synthase deficiency.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Enrolling by invitation</last_known_status>
  <start_date>January 2014</start_date>
  <completion_date type="Anticipated">December 2020</completion_date>
  <primary_completion_date type="Anticipated">December 2020</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>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Body length compared to normal pediatric growth curves.</measure>
    <time_frame>Quarterly measures- change from baseline over 24 months.</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change in scores of standardized developmental assessments (Vineland &amp; Batelle)</measure>
    <time_frame>Quarterly measures - change from baseline over 24 months</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Body Weight compared to normal pediatric growth curves</measure>
    <time_frame>Quarterly measurements from baseline over 24 months</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Head circumference compared to normal pediatric growth curves.</measure>
    <time_frame>Quarterly measures from baseline over 24 months</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Concentration of ganglioside GM3 in blood plasma</measure>
    <time_frame>Six times per year over 24 months</time_frame>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">20</enrollment>
  <condition>GM3 Synthase Deficiency</condition>
  <arm_group>
    <arm_group_label>Supplementation of gangliosides</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Supplementation of dairy-derived concentrated gangliosides</description>
  </arm_group>
  <intervention>
    <intervention_type>Dietary Supplement</intervention_type>
    <intervention_name>Supplementation of dairy-derived concentrated gangliosides.</intervention_name>
    <arm_group_label>Supplementation of gangliosides</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Clinical diagnosis of GM3 synthase deficiency&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        -&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>20 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Heng Wang, MD PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>DDC Clinic</affiliation>
  </overall_official>
  <verification_date>February 2019</verification_date>
  <study_first_submitted>September 3, 2014</study_first_submitted>
  <study_first_submitted_qc>September 8, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">September 9, 2014</study_first_posted>
  <last_update_submitted>February 6, 2019</last_update_submitted>
  <last_update_submitted_qc>February 6, 2019</last_update_submitted_qc>
  <last_update_posted type="Actual">February 7, 2019</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <!-- 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/NCT02234024
org study id: DDC-62314
nct id: NCT02234024
lead sponsor: 
has dmc: No
The purpose of this pilot project is to see if a supplemental form of dietary gangliosides
      can serve as a potential treatment for the rare metabolic condition called ganglioside GM3
      synthase deficiency.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Body length compared to normal pediatric growth curves.
time frame: Quarterly measures- change from baseline over 24 months.
measure: Change in scores of standardized developmental assessments (Vineland & Batelle)
time frame: Quarterly measures - change from baseline over 24 months
measure: Body Weight compared to normal pediatric growth curves
time frame: Quarterly measurements from baseline over 24 months
measure: Head circumference compared to normal pediatric growth curves.
time frame: Quarterly measures from baseline over 24 months
measure: Concentration of ganglioside GM3 in blood plasma
time frame: Six times per year over 24 months
arm group label: Supplementation of gangliosides
arm group type: Experimental
description: Supplementation of dairy-derived concentrated gangliosides
intervention type: Dietary Supplement
intervention name: Supplementation of dairy-derived concentrated gangliosides.
arm group label: Supplementation of gangliosides
criteria: 
gender: All
minimum age: N/A
maximum age: 20 Years
healthy volunteers: No
last name: Heng Wang, MD PhD
role: Principal Investigator
affiliation: DDC Clinic
responsible party type: Sponsor