Oral Supplementation of Gangliosides to Treat a Rare Metabolic Disorder

NCT ID: NCT02234024

Last Updated: 2019-02-07

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

NA

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-01-31

Study Completion Date

2020-12-31

Brief Summary

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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

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Conditions

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GM3 Synthase Deficiency

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Supplementation of gangliosides

Supplementation of dairy-derived concentrated gangliosides

Group Type EXPERIMENTAL

Supplementation of dairy-derived concentrated gangliosides.

Intervention Type DIETARY_SUPPLEMENT

Interventions

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Supplementation of dairy-derived concentrated gangliosides.

Intervention Type DIETARY_SUPPLEMENT

Eligibility Criteria

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Inclusion Criteria

* Clinical diagnosis of GM3 synthase deficiency

Exclusion Criteria

\-
Maximum Eligible Age

20 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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DDC Clinic - Center for Special Needs Children

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Heng Wang, MD PhD

Role: PRINCIPAL_INVESTIGATOR

DDC Clinic

Other Identifiers

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DDC-62314

Identifier Type: -

Identifier Source: org_study_id

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