Oral Supplementation of Gangliosides to Treat a Rare Metabolic Disorder

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

Sponsors

Lead Sponsor: DDC Clinic - Center for Special Needs Children

Source DDC Clinic - Center for Special Needs Children
Brief Summary

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.

Overall Status Enrolling by invitation
Start Date January 2014
Completion Date December 2020
Primary Completion Date December 2020
Phase N/A
Study Type Interventional
Primary Outcome
Measure Time Frame
Body length compared to normal pediatric growth curves. Quarterly measures- change from baseline over 24 months.
Change in scores of standardized developmental assessments (Vineland & Batelle) Quarterly measures - change from baseline over 24 months
Body Weight compared to normal pediatric growth curves Quarterly measurements from baseline over 24 months
Head circumference compared to normal pediatric growth curves. Quarterly measures from baseline over 24 months
Secondary Outcome
Measure Time Frame
Concentration of ganglioside GM3 in blood plasma Six times per year over 24 months
Enrollment 20
Condition
Intervention

Intervention Type: Dietary Supplement

Intervention Name: Supplementation of dairy-derived concentrated gangliosides.

Arm Group Label: Supplementation of gangliosides

Eligibility

Criteria:

Inclusion Criteria: - Clinical diagnosis of GM3 synthase deficiency Exclusion Criteria: -

Gender: All

Minimum Age: N/A

Maximum Age: 20 Years

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Heng Wang, MD PhD Principal Investigator DDC Clinic
Verification Date

February 2019

Responsible Party

Type: Sponsor

Has Expanded Access No
Number Of Arms 1
Arm Group

Label: Supplementation of gangliosides

Type: Experimental

Description: Supplementation of dairy-derived concentrated gangliosides

Study Design Info

Allocation: N/A

Intervention Model: Single Group Assignment

Primary Purpose: Treatment

Masking: None (Open Label)

Source: ClinicalTrials.gov