Oral Supplementation of Gangliosides to Treat a Rare Metabolic Disorder

DDC Clinic - Center for Special Needs Children20 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

GM3 Synthase Deficiency

Interventions

Supplementation of dairy-derived concentrated gangliosides.DIETARY_SUPPLEMENT

Eligibility

Sex: ALLMax age: 20 Years

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

Body length compared to normal pediatric growth curves.

Time frame: 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

Body Weight compared to normal pediatric growth curves

Time frame: Quarterly measurements from baseline over 24 months

Head circumference compared to normal pediatric growth curves.

Time frame: Quarterly measures from baseline over 24 months

Secondary Outcomes

Concentration of ganglioside GM3 in blood plasma

Time frame: Six times per year over 24 months

Data from ClinicalTrials.gov

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