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A Natural History Study of the Gangliosidoses

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This study's primary aims are to define and characterize disease progression for the infantile and juvenile forms of the gangliosidoses, and the late-onset forms of gangliosidosis, including their heterogeneity; and to observe treatment outcomes for any treatments tried. The secondary aims of this study are to understand the neurological involvement in late-onset gangliosidosis; and to collect data on disease progression that can be used for creation of an objective disease stage and severity index.

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Not specified
This study is NOT accepting healthy volunteers
Inclusion Criteria:

• 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.
Exclusion Criteria:

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

Tay-Sachs Disease, Sandhoff Disease, Late Onset Tay-Sachs Disease, GM1 Gangliosidosis, GM2 Gangliosidosis

Tay-Sachs disease, Sandhoff disease, Late Onset Tay-Sachs disease, LOTS, hexosaminidase A deficiency, hexosaminidase A and B deficiency, infantile Tay-Sachs disease, adult-onset Tay-Sachs disease, prospective, natural history, GM1 gangliosidosis, gangliosidoses, β-galactosidase, β-galactosidase deficiency, hexosaminidase, hexosaminidase deficiency, Tay-Sachs, Sandhoff, juvenile Tay-Sachs, juvenile Tay-Sachs disease, late onset Tay-Sachs, juvenile Sandhoff, juvenile Sandhoff disease, GM2 gangliosidosis

Jeanine Jarnes - utzx0002@umn.edu
Jeanine Jarnes
N/A
1007M85712
NCT00668187
See this study on ClinicalTrials.gov

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