New gene therapy targets rare, aggressive ALS

NCT ID NCT06100276

Ongoing Disease control Sponsor: UniQure Biopharma B.V. Source: ClinicalTrials.gov ↗

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

Summary

This early-stage trial tests a gene therapy called AMT-162 in 20 adults with a specific genetic form of ALS (SOD1-ALS). The treatment is given as a single injection into the spinal fluid. The main goal is to check safety and tolerability, while also looking for early signs that it might slow the disease. Participants must have a confirmed SOD1 gene mutation and still have at least moderate physical function.

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Conditions

The condition(s) this trial relates to.

amyotrophic lateral sclerosis amyotrophic lateral sclerosis type 1

As listed by the trial registrant

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

AMYOTROPHIC LATERAL SCLEROSIS

Contacts and locations

Locations

Barrow Neurological Institute

Phoenix, Arizona, 85013, United States
California Pacific Medical Center

San Francisco, California, 94109, United States
Columbia University Irving Medical Center

New York, New York, 10032, United States
Massachusetts General Hospital, Sean M. Healey and AMG Center for ALS Research

Boston, Massachusetts, 02114, United States
Mayo Clinic Florida

Jacksonville, Florida, 32224, United States
Mayo Clinic Rochester

Rochester, Minnesota, 55905, United States
Norrlands Universitetssjukhus

Umeå, Vasterbottens Ian, Sweden
Northwestern University Feinberg School of Medicine

Chicago, Illinois, 60611, United States
University of California Irvine

Irvine, California, 92697, United States
University of Kansas Medical Center

Fairway, Kansas, 66205, United States
University of Pennsylvania School of Medicine

Philadelphia, Pennsylvania, 19104, United States
Winship Cancer Institute of Emory University

Atlanta, Georgia, 30322, United States

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