Understanding the progression of gangliosidosis diseases

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study aims to characterize and describe the disease progression and heterogeneity of gangliosidosis diseases, including Tay-Sachs disease, Sandhoff disease, and GM1 gangliosidosis. By developing a quantitative method to delineate disease progression, the study seeks to provide essential data for evaluating future treatments, such as gene therapy. Longitudinal data will be collected from individuals diagnosed with these conditions to better understand their natural history and inform medical decisions. The findings will also help in providing objective measurements of treatment outcomes and guiding parents regarding potential outcomes for their children.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Subjects must have a documented gangliosidosis disease.
2. Subjects must be able to complete appropriate neuropsychological and neurobehavioral assessments.
3. Late-onset gangliosidosis subjects must be able to tolerate a head MRI.

Exclusion Criteria:

1\. There are no exclusion criteria, beyond a desire not to participate.

Where it's running

• University of Minnesota - Pediatric Genetics and Metabolism
  Minneapolis, Minnesota, United States Recruiting

Study contacts

• Principal investigator: Jeanine R. Jarnes, PharmD — University of Minnesota - Fairview
• Study coordinator: Jeanine R. Jarnes, PharmD
• Email: utzx0002@umn.edu
• Phone: 612-626-5131

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.