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Scientists track rare brain diseases to pave way for future cures

NCT ID NCT00668187

Knowledge-focused Sponsor: University of Minnesota Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study follows children and adults with Tay-Sachs, Sandhoff, and GM1 gangliosidosis to understand how these diseases progress. Researchers will measure changes in motor skills, communication, and behavior each year. The goal is to create a clear picture of the diseases so future treatments, like gene therapy, can be tested effectively.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

What this could lead to
If successful, this study will provide crucial data to design future therapies and clinical trials for these rare diseases.
What could go wrong
This is an observational study, not a treatment trial. It does not test any intervention, so there is no direct benefit to participants.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 52 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2010

Expected to finish

Mar 2027

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Any infant or juvenile with Tay-Sachs disease, Sandhoff disease, or GM1 gangliosidosis; and any adult with a late-onset gangliosidosis disease

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

Gangliosidoses GM1 gangliosidosis GM2 gangliosidosis Sandhoff disease Tay-Sachs disease Tay-Sachs disease, B variant, adult form Tay-Sachs Disease, Juvenile

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

GM1 gangliosidosis GM2 gangliosidosis Late onset Tay-Sachs disease Sandhoff disease Tay-Sachs disease

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • University of Minnesota - Pediatric Genetics and Metabolism

    RECRUITING

    Minneapolis, Minnesota, 55455, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.