Oral Supplementation of Gangliosides to Treat a Rare Metabolic Disorder

February 6, 2019 updated by: DDC Clinic - Center for Special Needs Children

Oral Supplementation of Gangliosides - A Potential Treatment for GM3 Synthase Deficiency

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.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

20

Phase

  • Not Applicable

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Clinical diagnosis of GM3 synthase deficiency

Exclusion Criteria:

-

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Supplementation of gangliosides
Supplementation of dairy-derived concentrated gangliosides
Dietary supplement: Supplementation of dairy-derived concentrated gangliosides.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Body length compared to normal pediatric growth curves.
Time Frame: Quarterly measures- change from baseline over 24 months.
Quarterly measures- change from baseline over 24 months.
Change in scores of standardized developmental assessments (Vineland & Batelle)
Time Frame: Quarterly measures - change from baseline over 24 months
Quarterly measures - change from baseline over 24 months
Body Weight compared to normal pediatric growth curves
Time Frame: Quarterly measurements from baseline over 24 months
Quarterly measurements from baseline over 24 months
Head circumference compared to normal pediatric growth curves.
Time Frame: Quarterly measures from baseline over 24 months
Quarterly measures from baseline over 24 months

Secondary Outcome Measures

Outcome Measure
Time Frame
Concentration of ganglioside GM3 in blood plasma
Time Frame: Six times per year over 24 months
Six times per year over 24 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

DDC Clinic - Center for Special Needs Children

Investigators

  • Principal Investigator: Heng Wang, MD PhD, DDC Clinic

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2014

Primary Completion (Anticipated)

December 1, 2020

Study Completion (Anticipated)

December 1, 2020

Study Registration Dates

First Submitted

September 3, 2014

First Submitted That Met QC Criteria

September 8, 2014

First Posted (Estimate)

September 9, 2014

Study Record Updates

Last Update Posted (Actual)

February 7, 2019

Last Update Submitted That Met QC Criteria

February 6, 2019

Last Verified

February 1, 2019

More Information

Terms related to this study

Other Study ID Numbers

  • DDC-62314

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on GM3 Synthase Deficiency

Clinical Trials on Supplementation of dairy-derived concentrated gangliosides.

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