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A Natural History Study of the Gangliosidoses

Who can join

All ages · All sexes

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 exclusion criteria, beyond a desire not to participate.

About the study

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.

Sponsor: University of Minnesota · Participants: 52 · Started: 2010-12

Contact the study team

• Jeanine R. Jarnes, PharmD · Phone: 612-626-5131

Official record on ClinicalTrials.gov — NCT00668187

Locations in the U.S.

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.