Oral Supplementation of Gangliosides to Treat a Rare Metabolic Disorder

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

Status

UNKNOWN

Phases

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT02234024

Enrollment

Registered

2014-09-09

Start date

2014-01-31

Completion date

2020-12-31

Last updated

2019-02-07

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

GM3 Synthase Deficiency

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.

Interventions

DIETARY_SUPPLEMENTSupplementation of dairy-derived concentrated gangliosides.

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

No minimum to 20 Years

Healthy volunteers

Inclusion criteria

* Clinical diagnosis of GM3 synthase deficiency

Exclusion criteria

Design outcomes

Primary

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

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

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026