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A Natural History Study of the Gangliosidoses

Recruitingn/aAges any - anyAllUniversity of Minnesota
NCT00668187 on ClinicalTrials.gov ↗
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In plain words

What the trial is, and whether it could suit you

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.

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📋 Eligibility rules (as published)
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.
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What it tests

  • Not listed
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Where

1 site
University of Minnesota - Pediatric Genetics and Metabolism
Minneapolis, United States
🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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