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A Natural History Study of the Gangliosidoses

NCT00668187 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 52

Last updated 2026-03-04

No results posted yet for this study

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.

Conditions

  • Tay-Sachs Disease
  • Sandhoff Disease
  • Late Onset Tay-Sachs Disease
  • GM1 Gangliosidosis
  • GM2 Gangliosidosis

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

  • Lysosomal Disease Network

    collaborator OTHER

  • University of Minnesota

    lead OTHER

Principal Investigators

  • Jeanine R. Jarnes, PharmD · University of Minnesota - Fairview

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2010-12-31
Primary Completion
2027-03-01
Completion
2027-03-01

Countries

  • United States

Study Locations

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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 NCT00668187 on ClinicalTrials.gov