MedTrace Logo

Gene Therapy for Tay-Sachs Disease

NCT01869270 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 4

Last updated 2014-12-04

No results posted yet for this 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

  • Tay Sachs Disease
  • Sandhoff Disease
  • Late Onset Tay Sachs Disease

Sponsors & Collaborators

  • Rare Diseases Clinical Research Network

    collaborator NETWORK

  • National Center for Advancing Translational Sciences (NCATS)

    collaborator NIH

  • National Institute of Neurological Disorders and Stroke (NINDS)

    collaborator NIH

  • National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

    collaborator NIH

  • University of Minnesota

    lead OTHER

Principal Investigators

  • Chester B. Whitley, PhD, MD · University of Minnesota

  • Jeffrey Krischer, PhD · University of South Florida

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2010-12-31
Primary Completion
2014-07-31
Completion
2014-08-31

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01869270 on ClinicalTrials.gov