Gene therapy injection tested for ALS safety
NCT ID NCT02039401
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This early-phase trial tested whether a gene therapy called VM202 is safe when injected into muscles of 18 people with ALS. The study focused on side effects over 9 months, not on whether the treatment improves symptoms. All participants had ALS for less than 2 years and had at least moderate lung function.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
VM202 (a gene therapy given as injections into muscles)
What this could lead to
If safe, this could pave the way for larger studies testing whether VM202 slows muscle decline in ALS.
What could go wrong
This is a very early, small trial (18 people) focused only on safety. It cannot prove whether VM202 helps ALS symptoms or changes the disease course.
Disclaimer
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This is a summary of
the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
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Locations
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Northwestern University
Chicago, Illinois, 60611, United States