Oral Supplementation of Gangliosides to Treat a Rare Metabolic Disorder

Not Applicable

• Conditions: GM3 Synthase Deficiency

• Registration Number: NCT02234024
• Lead Sponsor: 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2014-01
• Study First Submitted: 2014-09-03
• Study First Submitted QC: 2014-09-08
• Study First Posted: 2014-09-09
• Status Verified: 2019-02
• Last Update Submitted: 2019-02-06
• Last Update Posted: 2019-02-07
• Primary Completion: 2020-12
• Study Completion: 2020-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

• Clinical diagnosis of GM3 synthase deficiency

Exclusion Criteria

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
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
Body length compared to normal pediatric growth curves.	Quarterly measures- change from baseline over 24 months.

Secondary Outcome Measures

Name	Time	Method
Concentration of ganglioside GM3 in blood plasma	Six times per year over 24 months