GM3 Synthase Deficiency Clinical Trial
Official title:
Oral Supplementation of Gangliosides - A Potential Treatment for GM3 Synthase Deficiency
| Verified date | February 2019 |
| Source | DDC Clinic - Center for Special Needs Children |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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.
| Status | Enrolling by invitation |
| Enrollment | 20 |
| Est. completion date | December 2020 |
| Est. primary completion date | December 2020 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | N/A to 20 Years |
| Eligibility |
Inclusion Criteria: - Clinical diagnosis of GM3 synthase deficiency Exclusion Criteria: |
| Country | Name | City | State |
|---|---|---|---|
| n/a | |||
| Lead Sponsor | Collaborator |
|---|---|
| DDC Clinic - Center for Special Needs Children |
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Body length compared to normal pediatric growth curves. | Quarterly measures- change from baseline over 24 months. | ||
| Primary | Change in scores of standardized developmental assessments (Vineland & Batelle) | Quarterly measures - change from baseline over 24 months | ||
| Primary | Body Weight compared to normal pediatric growth curves | Quarterly measurements from baseline over 24 months | ||
| Primary | Head circumference compared to normal pediatric growth curves. | Quarterly measures from baseline over 24 months | ||
| Secondary | Concentration of ganglioside GM3 in blood plasma | Six times per year over 24 months |