Oral Supplementation of Gangliosides to Treat a Rare Metabolic Disorder

DDC Clinic - Center for Special Needs Children

Status: Unknown

Conditions: GM3 Synthase Deficiency

Treatments: Dietary Supplement: Supplementation of dairy-derived concentrated gangliosides.

Study type: Interventional

Funder types: Other

Identifiers

NCT02234024

DDC-62314

Details and patient eligibility

About

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.

Enrollment: 20 estimated patients

Sex: All

Ages: Under 20 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Clinical diagnosis of GM3 synthase deficiency

Exclusion criteria

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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20 participants in 1 patient group

Supplementation of gangliosides

Experimental group

Description:

Supplementation of dairy-derived concentrated gangliosides

Treatment:

Dietary Supplement: Supplementation of dairy-derived concentrated gangliosides.