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New ALS drug targets nerve damage in early human trial

NCT ID NCT06665165

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.
Why investors are watching

Amylyx Pharmaceuticals is testing AMX0114, an antisense oligonucleotide drug, in a small Phase 1 trial for ALS. For a small company, this early safety and tolerability readout matters because it is the first test of a new drug candidate in people, and the result will shape the company's pipeline and future spending.

If it works: If the drug proves safe and tolerable in this 48-person study, Amylyx could advance AMX0114 to larger trials, adding a second potential ALS treatment to its portfolio. A positive readout may also reassure investors that the company has a viable research program beyond its existing products.

If it fails: Phase 1 trials often fail because the drug causes unexpected side effects or does not behave as expected in the body. A negative result or a delay would likely force Amylyx to reassess its pipeline and could hurt investor confidence, since the company's value depends heavily on its few drug candidates.

AI-written from the trial record. Speculative, and not investment advice.

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

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 48 people

The number the study aims to enrol. It can still change while the study runs.

Started

Apr 2025

Expected to finish

Oct 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Ability to understand the purpose and risks of this study, willingness to comply with the study and to provide informed consent in accordance with local laws and regulations. 2. Male or female, at least 18 years of age. 3. Diagnosis of clinically definite or clinically probable ALS, made by a physician who is experienced with management of ALS. 4. Time since onset of first symptom of ALS should be \<24 months prior to beginning the study. Date of ALS symptom onset is defined as the onset of weakness (in the limbs, bulbar region, or trunk). 5. If the participant is to be treated with riluzole and/or edaravone before or during the trial, then treatment must be previously started and maintained at a stable regimen for at least 30 days prior to starting the study and through the end of the study. 6. Women of childbearing potential (e.g., not post-menopausal for at least one year or surgically sterile) must agree to use an acceptable birth control method for the duration of the trial and 60 days after the last dose of Study Drug or be of non-childbearing potential. 7. Female participants or female partners of male participants must not be pregnant or plan to become pregnant for the duration of the trial and for up to 90 days after the last dose of Study Drug. 8. Male participants must agree to abstain from sperm donation for the duration of the trial and practice contraception with a female partner, for at least 90 days after last dose of Study Drug. Exclusion Criteria: 1. Presence of tracheostomy or permanent assisted ventilation. 2. SVC less than 65%. 3. Abnormal liver function defined as aspartate aminotransferase and/or alanine aminotransferase \> 3 times the upper limit of normal (ULN) and/or total bilirubin \> 1.5 times the ULN (obtained within 4 weeks of first dose) except when a result of Gilbert syndrome. 4. Abnormal renal function defined as estimated glomerular filtration rate (eGFR) \< 60 mL/min/1.73m2. 5. Other laboratory abnormalities, including abnormalities in platelet count, international normalized ratio, prothrombin time, and activated partial thromboplastin time. 6. Pregnant women (confirmed by a pregnancy test within 7 days prior to first dose) or women currently breastfeeding. 7. Current or previous clinically significant, unstable medical condition (other than ALS), that in the opinion of the Investigator could affect a participant's safety or ability to comply with the study. 8. Significant abnormalities in physical/neurological examination, vital signs, or electrocardiogram (ECG), which in the opinion of the Investigator could affect the safety of the participant. 9. Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that could affect the participant's ability to provide informed consent or comply with study procedures. 10. Current or previous enrollment in another trial involving use of an investigational therapy, in most cases within 30 days after the last dose of the study drug, prior to starting this study. 11. Current or previous treatment with small interfering ribonucleic acid, stem cell therapy, any ASO or gene therapy. 12. Any contraindications for lumbar puncture or repeated intrathecal injection and/or underlying disorders that could be affected by intrathecal injections. 13. Prior severe reaction or known hypersensitivity to any part of the Study Drug.

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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    14 sites in 2 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

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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