ALS drug already approved for some may help others

NCT ID NCT07294144

Recruiting now Disease control Sponsor: Washington University School of Medicine Source: ClinicalTrials.gov ↗

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

Summary

This phase 2 trial tests whether tofersen, a drug already approved for a specific type of ALS, can help people with ALS who do not have the SOD1 gene mutation. Thirty adults will receive the drug via spinal injections over 24 weeks. Researchers will measure changes in nerve damage markers and monitor safety.

What this could mean

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

Active substance: tofersen
What this could lead to: If successful, this could expand the use of tofersen to a broader group of ALS patients who lack the SOD1 mutation, potentially slowing disease progression.
What could go wrong: This is a small, early-phase trial (30 people) with no placebo group, so results may not be definitive. Tofersen is already approved for SOD1-ALS, but its effect on non-SOD1 ALS is uncertain.

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.

amyotrophic lateral sclerosis

As listed by the trial registrant

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

ALS - AMYOTROPHIC LATERAL SCLEROSIS

Contacts and locations

Locations

Emory University
RECRUITING

Atlanta, Georgia, 30322, United States
Massachusetts General Hospital
RECRUITING

Boston, Massachusetts, 02114, United States
Washington University ALS Center
RECRUITING

St Louis, Missouri, 63110, United States

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