A Natural History Study of the Gangliosidoses
Recruiting
Observational Study
Tay-Sachs Disease
Sandhoff Disease
Late Onset Tay-Sachs Disease
GM1 Gangliosidosis
GM2 Gangliosidosis
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Sex
- Any
- Study type
- Observational
- Participants needed
- 52 (estimated)
- Sponsor
- University of Minnesota · Other
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About This Trial
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 progression for the gangliosidosis diseases (Tay-Sachs disease, Sandhoff disease, and GM1 gangliosidosis) in order to better understand the natural history and heterogeneity of these diseases. Such a quantita…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
1. Subjects must have a documented gangliosidosis disease.
2. Subjects must be able to complete appropriate neuropsychological and neurobehavioral assessments.
3. Late-onset gangliosidosis subjects must be able to tolerate a head MRI.
Exclusion Criteria:
1\. There are no exclu…
Contacts