Actively Recruiting
A Natural History Study of the Gangliosidoses
Led by University of Minnesota · Updated on 2026-03-04
52
Participants Needed
1
Research Sites
847 weeks
Total Duration
On this page
Sponsors
U
University of Minnesota
Lead Sponsor
R
Rare Diseases Clinical Research Network
Collaborating Sponsor
AI-Summary
What this Trial Is About
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 quantitative method will also be essential for evaluating any treatments that may become available in the future, such as gene therapy. The data from this study will be necessary to provide end-points for future therapies, guide medical decisions about treatment, provide objective measurement of treatment outcomes, and accurately inform parents regarding potential outcomes.
CONDITIONS
Official Title
A Natural History Study of the Gangliosidoses
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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.
You will not qualify if you...
- No exclusion criteria except a desire not to participate.
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
University of Minnesota - Pediatric Genetics and Metabolism
Minneapolis, Minnesota, United States, 55455
Actively Recruiting
Research Team
J
Jeanine R. Jarnes, PharmD
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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