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NCT01869270 · ClinicalTrials.gov registry record
Gene Therapy for Tay-Sachs Disease
A clinical trial of Tay-Sachs Disease and Sandhoff Disease, sponsored by University of Minnesota.
- Completed
- Registry status
- 4
- Enrollment target
- 2
- Study locations
NCT01869270 is a study of Tay-Sachs Disease and Sandhoff Disease that has completed, run by University of Minnesota. The registered enrollment target is 4 participants, below the 327-participant average among 6 other Tay-Sachs Disease trials with a reported enrollment target (99% lower). The trial reports 2 study locations across 2 states.
The verdict
NCT01869270, a study of Tay-Sachs Disease and Sandhoff Disease, has completed, sponsored by University of Minnesota.
- COMPLETED
- Registry status
- 4 participants
- Enrollment target
- 2
- Study locations
Study 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 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.
Conditions Studied
Study Locations (2)
Florida
- Data Management and Coordinating Center (DMCC), Univ. of South Florida - Tampa
Minnesota
- University of Minnesota - Minneapolis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 4 participants |
| Start Date | 2010-12 |
| Est. Completion | 2014-08 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01869270
The ClinicalTrials.gov registry entry for NCT01869270 describes a study currently listed as completed, categorized as an unspecified phase. The registered enrollment target is 4 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 327-participant average among 6 other Tay-Sachs Disease trials with a reported enrollment target (99% lower). The listed sponsor is University of Minnesota, which has 966 total studies on file at ClinicalTrials.gov.
The record links to 3 conditions, with Tay-Sachs Disease appearing as the primary indexed condition, and to 0 interventions.
NCT01869270 reports 2 study locations spanning 2 distinct geographic areas - top geographies include Florida, Minnesota.
Frequently Asked Questions
What is clinical trial NCT01869270 about?
NCT01869270 is a clinical study titled "Gene Therapy for Tay-Sachs Disease". 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 max...
What is the current status of trial NCT01869270?
This trial is currently completed. The enrollment target is 4 participants. The study started on 2010-12. Estimated completion is 2014-08.
What conditions does trial NCT01869270 study?
This clinical trial studies the following conditions: Tay-Sachs Disease, Sandhoff Disease, Late Onset Tay-Sachs Disease.
Who is sponsoring clinical trial NCT01869270?
This trial is sponsored by University of Minnesota, which has 966 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01869270 being conducted?
This trial has 2 study locations across Florida, Minnesota. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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Read our methodology - how this data is sourced, computed, and verified.
Related
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