RecruitingPhase 1NCT06665165

AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis

Phase 1, Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose Study to Evaluate Safety, Tolerability, PK and PD of Antisense Oligonucleotide AMX0114 Administered to Adult Participants With Amyotrophic Lateral Sclerosis


Sponsor

Amylyx Pharmaceuticals Inc.

Enrollment

48 participants

Start Date

Apr 7, 2025

Study Type

INTERVENTIONAL

Conditions

Summary

This study is a placebo-controlled Phase I study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of the antisense oligonucleotide (ASO) AMX0114 in adult participants with amyotrophic lateral sclerosis (ALS).


Eligibility

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This study is testing the safety and effects of a new investigational drug called AMX0114 in people with ALS (amyotrophic lateral sclerosis, also known as Lou Gehrig's disease), a progressive disease that damages nerve cells controlling muscle movement. **You may be eligible if...** - You are 18 or older - You have been diagnosed with clinically definite or clinically probable ALS by an experienced specialist - Your first ALS symptom (muscle weakness) began less than 24 months ago - If you are taking riluzole or edaravone (standard ALS treatments), you have been on a stable dose for at least 30 days - You agree to use contraception during the study if you could become pregnant or father a child **You may NOT be eligible if...** - You have had ALS symptoms for more than 24 months - You have serious organ function problems unrelated to ALS - You are pregnant or planning to become pregnant during the trial - Your disease is too advanced for the treatment to be safely administered Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

Interested in this trial?

Get notified about updates and connect with the research team.

Interventions

DRUGAMX0114

Antisense oligonucleotides (ASOs) are a type of medicine that treats diseases by intercepting the mRNA messages sent within the cell, resulting in fewer specific proteins being made. AMX0114 is an ASO that targets the mRNA messenger that instructs the body to create a protein called calpain-2. Calpain-2 has been linked to the degeneration and death of neurons in many neurological diseases, including people living with sporadic ALS. AMX0114 is designed to reduce the levels of calpain-2, with the goal of slowing down the process that leads to neuron injury and death.

OTHERPlacebo

Placebo


Locations(14)

University of California, San Diego

La Jolla, California, United States

Georgetown University Hospital Pasquerilla Healthcare Center

Washington D.C., District of Columbia, United States

University of Florida

Gainesville, Florida, United States

Mayo Clinic in Florida

Jacksonville, Florida, United States

Orlando Regional Medical Center, Orlando Health Neuroscience Institute

Orlando, Florida, United States

Massachusetts General Hospital, Healey & AMG Center for ALS

Boston, Massachusetts, United States

Mayo Clinic in Rochester

Rochester, Minnesota, United States

Temple University of the Commonwealth System of Higher Education

Philadelphia, Pennsylvania, United States

Alliance for Multispecialty Research, LLC

Knoxville, Tennessee, United States

Houston Methodist Neurological Institute

Houston, Texas, United States

University of Calgary

Calgary, Alberta, Canada

McMaster University

Hamilton, Ontario, Canada

London Health Sciences Centre

London, Ontario, Canada

McGill University Health Centre - Centre for Innovative Medicine

Montreal, Quebec, Canada

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

Visit

NCT06665165


Related Trials