AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis
Start Date
4/7/2025
Completion Date
10/1/2027
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).
Detailed Description
The purpose of this study is to determine how safe and tolerable the investigational drug, AMX0114, is for the treatment of amyotrophic lateral sclerosis (ALS). AMX0114 is given by intrathecal injection, an injection in the lower back into the spinal canal, also known as lumbar puncture. This clinical trial is designed to test if the treatment is safe and tolerable by monitoring the incidence of adverse events, serious adverse events, dose limiting toxicities (DLTs), and incidence of abnormalities in clinical laboratory assessments, vital signs, physical and neurological examinations, and electrocardiograms (ECGs). This trial will also assess the effects of AMX0114 on biomarkers of ALS, including markers of neuronal death and neuroinflammation.
Eligibility Criteria
Age Range: 18 years to No maximum
Interventions
AMX0114
Placebo
Conditions
Locations
University of California, San Diego
La Jolla, California 92093
United States
Georgetown University Hospital Pasquerilla Healthcare Center
Washington D.C., District of Columbia 20007
United States
University of Florida
Gainesville, Florida 32611
United States
Mayo Clinic in Florida
Jacksonville, Florida 32224
United States
Orlando Regional Medical Center, Orlando Health Neuroscience Institute
Orlando, Florida 32806
United States
Massachusetts General Hospital, Healey & AMG Center for ALS
Boston, Massachusetts 02114
United States
Mayo Clinic in Rochester
Rochester, Minnesota 55905
United States
Temple University of the Commonwealth System of Higher Education
Philadelphia, Pennsylvania 19140
United States
Alliance for Multispecialty Research, LLC
Knoxville, Tennessee 37909
United States
Houston Methodist Neurological Institute
Houston, Texas 77030
United States
University of Calgary
Calgary, Alberta T2N 4Z6
Canada
McMaster University
Hamilton, Ontario L8N 3Z5
Canada
London Health Sciences Centre
London, Ontario N6G 2M3
Canada
McGill University Health Centre - Centre for Innovative Medicine
Montreal, Quebec H4A 3J1
Canada