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.

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

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for AMYOTROPHIC LATERAL SCLEROSIS are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

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

  • Northwestern University

    Chicago, Illinois, 60611, United States

More trials for these conditions

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