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.
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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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