Status:
COMPLETED
Gene Therapy for Tay-Sachs Disease
Lead Sponsor:
University of Minnesota
Collaborating Sponsors:
Rare Diseases Clinical Research Network
National Center for Advancing Translational Sciences (NCATS)
Conditions:
Tay Sachs Disease
Sandhoff Disease
Eligibility:
All Genders
Brief Summary
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 Hexo...
Detailed Description
Much has been done in the past four decades to better understand, improve diagnostic measures of, and prevent hexosaminidase deficiency diseases, yet all of them - Tay-Sachs, Sandhoff, and Late Onset ...
Eligibility Criteria
Inclusion
- Any person who has been diagnosed with a hexosaminidase deficiency disease can be included in this study.
Exclusion
- The only exclusion criteria is a desire not to participate in this study.
Key Trial Info
Start Date :
December 1 2010
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
August 1 2014
Estimated Enrollment :
4 Patients enrolled
Trial Details
Trial ID
NCT01869270
Start Date
December 1 2010
End Date
August 1 2014
Last Update
December 4 2014
Active Locations (2)
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1
Data Management and Coordinating Center (DMCC), Univ. of South Florida
Tampa, Florida, United States, 33612
2
University of Minnesota
Minneapolis, Minnesota, United States, 55455