A Natural History Study of the Gangliosidoses

Part of paid clinical trials in Minneapolis, Minnesota.

Sponsor
University of Minnesota
Study ID
NCT00668187
Status
Recruiting
Apply to this trial

Conditions

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

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Study Details

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 Dates

Start date
Dec 31, 2010
Status verified
Mar 2026
Primary completion
Mar 1, 2027
Completion
Mar 1, 2027

Study Design

Enrollment
52 participants (estimated)

Arms

  • Arm: Gangliosidosis Diseases Study Population
    This study observes one cohort: 42 infantile or juvenile Tay-Sachs disease, Sandhoff disease, or GM1 gangliosidosis affected subjects; and 10 late-onset gangliosidosis disease affected subjects.

Primary Outcome Measure

Change in Child Developmental Status as Assessed by Neuropsychological Tests [ Time Frame: Upon enrollment; then at 12, 24, 36, 48 and 60 months ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
University of Minnesota - Pediatric Genetics and MetabolismMinneapolisMinnesota55455
Jeanine R. Jarnes, PharmD
[email protected]
612-626-5131
Jeanine R. Jarnes, PharmD (PRINCIPAL_INVESTIGATOR)
Chester B. Whitley, MD, PhD (SUB_INVESTIGATOR)

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University of Minnesota - Pediatric Genetics and Metabolism· Minneapolis, MN

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