A Natural History Study of the Gangliosidoses

Recruiting now

Conditions studied: Tay-Sachs Disease, Sandhoff Disease, Late Onset Tay-Sachs Disease, GM1 Gangliosidosis, GM2 Gangliosidosis

In brief

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.

Key facts

Study ID

NCT00668187

Run by

University of Minnesota

People needed

52

Starts

2010-12-01

Expected to finish

2027-03-01

Last updated by the study team

2026-03-04

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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 may not qualify if…

• There are no exclusion criteria, beyond a desire not to participate.

Where it is running

• University of Minnesota - Pediatric Genetics and Metabolism — Minneapolis, Minnesota, United States (enrolling)

Full record on ClinicalTrials.gov

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