New ALS drug targets nerve damage in early human trial
NCT ID NCT06665165
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.
Disclaimer
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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.
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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Alliance for Multispecialty Research, LLC
RECRUITINGKnoxville, Tennessee, 37909, United States
Contact Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Georgetown University Hospital Pasquerilla Healthcare Center
RECRUITINGWashington D.C., District of Columbia, 20007, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Houston Methodist Neurological Institute
RECRUITINGHouston, Texas, 77030, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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London Health Sciences Centre
RECRUITINGLondon, Ontario, N6G 2M3, Canada
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Massachusetts General Hospital, Healey & AMG Center for ALS
RECRUITINGBoston, Massachusetts, 02114, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Mayo Clinic in Florida
RECRUITINGJacksonville, Florida, 32224, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Mayo Clinic in Rochester
RECRUITINGRochester, Minnesota, 55905, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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McGill University Health Centre - Centre for Innovative Medicine
RECRUITINGMontreal, Quebec, H4A 3J1, Canada
Contact
Contact Email: •••••@•••••
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McMaster University
RECRUITINGHamilton, Ontario, L8N 3Z5, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Orlando Regional Medical Center, Orlando Health Neuroscience Institute
RECRUITINGOrlando, Florida, 32806, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Temple University of the Commonwealth System of Higher Education
RECRUITINGPhiladelphia, Pennsylvania, 19140, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Email: •••••@•••••
Contact
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University of Calgary
RECRUITINGCalgary, Alberta, T2N 4Z6, Canada
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of California, San Diego
RECRUITINGLa Jolla, California, 92093, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Florida
RECRUITINGGainesville, Florida, 32611, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••