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Late Onset Tay-Sachs Disease clinical trials

View clinical trials related to Late Onset Tay-Sachs Disease.

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NCT ID: NCT01869270 Completed - Sandhoff Disease Clinical Trials

Gene Therapy for Tay-Sachs Disease

Start date: December 2010
Phase: N/A
Study type: Observational

Hypothesis: To study the natural history of Tay-Sachs disease and evaluate therapeutic interventions. This study is intended to work in collaboration with NCT00668187 "A Natural History Study of Hexosaminidase Deficiency." Because so few patients with Tay-Sachs disease present annually, we will maximize both research projects by enrolling patients in both studies. For this present study, we will perform retrospective medical record review to gather data. Through this medical record review, we will collect biomarker analysis results, neuroimaging report data, quality-of-life questionnaire data and ophthalmology exam findings. If the subject has undergone therapy or treatment, the results will be noted.

NCT ID: NCT00668187 Recruiting - Sandhoff Disease Clinical Trials

A Natural History Study of the Gangliosidoses

Start date: December 2010
Phase:
Study type: Observational

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.