Gene Therapy for Tay-Sachs Disease

Completed

Conditions studied: Tay Sachs Disease, Sandhoff Disease, Late Onset Tay Sachs Disease

In brief

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.

Key facts

Study ID

NCT01869270

Run by

University of Minnesota

People needed

4

Starts

2010-12-01

Expected to finish

2014-08-01

Last updated by the study team

2014-12-04

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Any person who has been diagnosed with a hexosaminidase deficiency disease can be included in this study.

You may not qualify if…

• The only exclusion criteria is a desire not to participate in this study.

Where it is running

• Data Management and Coordinating Center (DMCC), Univ. of South Florida — Tampa, Florida, United States
• University of Minnesota — Minneapolis, Minnesota, United States

Full record on ClinicalTrials.gov

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