Gene therapy for rare brain diseases: 5-Year safety check begins
NCT ID NCT06614569
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study follows 7 people who previously received a gene therapy called AXO-AAV-GM2 for Tay-Sachs or Sandhoff disease — rare, fatal genetic disorders that destroy nerve cells. Researchers will track them for up to 5 years to see if the treatment remains safe and whether it helps slow the disease's natural course. The focus is on long-term side effects and changes in development, movement, and thinking skills.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- AXO-AAV-GM2 gene therapy
- What this could lead to
- If successful, this could show that a single gene therapy treatment can slow or stabilize the devastating progression of Tay-Sachs and Sandhoff diseases over several years.
- What could go wrong
- This is a very small, early-stage follow-up study with only 7 participants. It cannot prove the therapy works broadly, and there are risks like cancer or immune disorders that need long-term monitoring.
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
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About 7 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Sep 2024
- Expected to finish
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Dec 2028
An estimate. End dates often move.
- Lead sponsor
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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
Subjects with Tay-Sachs or Sandhoff Disease who were previously treated with AXO-AAV-GM2
- Ages
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Children (under 18), adults (18 to 64) and older adults (65 and over)
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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: * Subjects who were previously enrolled in the study "A Two-Stage Dose-Escalation Study to Evaluate the Safety and Efficacy of Bilateral Intraparenchymal thalamic and intracisternal/Intrathecal Admin of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease." Exclusion Criteria: * Subjects who were not previously enrolled under the initial protocol.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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UMass Chan Medical School
Worcester, Massachusetts, 01655, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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