New ALS drug targets nerve damage in early human trial

NCT ID NCT06665165

Recruiting now Disease control Sponsor: Amylyx Pharmaceuticals Inc. Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-stage trial tests a new medicine, AMX0114, in 48 adults with ALS. The drug aims to reduce a protein called calpain-2, which may damage nerves. The main goal is to check safety and how the body handles the drug, not yet to prove it works.

What this could mean

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

Active substance: AMX0114 (an antisense oligonucleotide designed to reduce calpain-2 protein levels)
What this could lead to: If successful, this could point toward a treatment that slows nerve damage in ALS by lowering calpain-2 levels.
What could go wrong: This is a very early Phase 1 trial with only 48 participants, focused on safety. It is too small and early to know if AMX0114 will work or be safe long-term.

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 motor neuron disorder

As listed by the trial registrant

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

ALS

Contacts and locations

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Study contacts

Contact

Email: •••••@•••••

Locations

Alliance for Multispecialty Research, LLC
RECRUITING

Knoxville, Tennessee, 37909, United States
Georgetown University Hospital Pasquerilla Healthcare Center
RECRUITING

Washington D.C., District of Columbia, 20007, United States
Houston Methodist Neurological Institute
RECRUITING

Houston, Texas, 77030, United States
London Health Sciences Centre
RECRUITING

London, Ontario, N6G 2M3, Canada
Massachusetts General Hospital, Healey & AMG Center for ALS
RECRUITING

Boston, Massachusetts, 02114, United States
Mayo Clinic in Florida
RECRUITING

Jacksonville, Florida, 32224, United States
Mayo Clinic in Rochester
RECRUITING

Rochester, Minnesota, 55905, United States
McGill University Health Centre - Centre for Innovative Medicine
RECRUITING

Montreal, Quebec, H4A 3J1, Canada
McMaster University
RECRUITING

Hamilton, Ontario, L8N 3Z5, Canada
Orlando Regional Medical Center, Orlando Health Neuroscience Institute
RECRUITING

Orlando, Florida, 32806, United States
Temple University of the Commonwealth System of Higher Education
RECRUITING

Philadelphia, Pennsylvania, 19140, United States
University of Calgary
RECRUITING

Calgary, Alberta, T2N 4Z6, Canada
University of California, San Diego
RECRUITING

La Jolla, California, 92093, United States
University of Florida
RECRUITING

Gainesville, Florida, 32611, United States

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