NCT00668187 A Natural History Study of the Gangliosidoses
| NCT ID | NCT00668187 |
| Status | Recruiting |
| Phase | — |
| Sponsor | University of Minnesota |
| Condition | Tay-Sachs Disease |
| Study Type | OBSERVATIONAL |
| Enrollment | 52 participants |
| Start Date | 2010-12 |
| Primary Completion | 2027-03-01 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 52 participants in total. It began in 2010-12 with a primary completion date of 2027-03-01.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
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.
Contact & Investigator
Jeanine R. Jarnes, PharmD
PRINCIPAL INVESTIGATOR
University of Minnesota - Fairview
Frequently Asked Questions
Who can join the NCT00668187 clinical trial?
This trial is open to participants of all sexes, studying Tay-Sachs Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT00668187 currently recruiting?
Yes, NCT00668187 is actively recruiting participants. Contact the research team at utzx0002@umn.edu for enrollment information.
Where is the NCT00668187 trial being conducted?
This trial is being conducted at Minneapolis, United States.
Who is sponsoring the NCT00668187 clinical trial?
NCT00668187 is sponsored by University of Minnesota. The principal investigator is Jeanine R. Jarnes, PharmD at University of Minnesota - Fairview. The trial plans to enroll 52 participants.