Status:
RECRUITING
A Natural History Study of the Gangliosidoses
Lead Sponsor:
University of Minnesota
Collaborating Sponsors:
Rare Diseases Clinical Research Network
National Center for Advancing Translational Sciences (NCATS)
Conditions:
Tay-Sachs Disease
Sandhoff Disease
Eligibility:
All Genders
Brief Summary
Hypothesis: To characterize and describe disease progression and heterogeneity of the gangliosidosis diseases. This research study seeks to develop a quantitative method to delineate disease progress...
Detailed Description
The infantile form of GM2 and GM1 gangliosidosis diseases ("classic" infantile) is the most common. Infants with Tay-Sachs disease, Sandhoff disease or GM1 gangliosidosis appear normal at birth, but a...
Eligibility Criteria
Inclusion
- Subjects must have a documented gangliosidosis disease.
- Subjects must be able to complete appropriate neuropsychological and neurobehavioral assessments.
- Late-onset gangliosidosis subjects must be able to tolerate a head MRI.
Exclusion
- 1\. There are no exclusion criteria, beyond a desire not to participate.
Key Trial Info
Start Date :
December 1 2010
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
March 1 2027
Estimated Enrollment :
52 Patients enrolled
Trial Details
Trial ID
NCT00668187
Start Date
December 1 2010
End Date
March 1 2027
Last Update
February 25 2025
Active Locations (1)
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1
University of Minnesota - Pediatric Genetics and Metabolism
Minneapolis, Minnesota, United States, 55455