Actively Recruiting
Phase 1, Randomized, Double-blind, Placebo-controlled Study to Evaluate Safety, Tolerability, and Pharmacokinetics of AMX0114 in Adults With Amyotrophic Lateral Sclerosis
Led by Amylyx Pharmaceuticals Inc. · Updated on 2026-04-14
48
Participants Needed
14
Research Sites
34 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of AMX0114, an investigational antisense oligonucleotide drug, in adults living with amyotrophic lateral sclerosis (ALS). This phase I, placebo-controlled study aims to monitor how the drug affects ALS by targeting a protein linked to nerve cell damage, with the goal of understanding its impact on disease markers and overall safety. The study involves two groups: one receiving AMX0114 and the other a placebo, both administered through intrathecal injection (injection into the spinal canal) once every 4 weeks for up to four doses on Days 1, 29, 57, and 85. Researchers will carefully observe participants throughout the dosing period to evaluate drug effects and potential side effects. Participants will undergo regular monitoring including physical and neurological exams, vital signs checks, laboratory tests, electrocardiograms, and biomarker assessments related to ALS progression. The primary focus is on safety and tolerability from Day 1 through Day 145. The study includes comprehensive evaluations to track drug behavior in the body and its influence on ALS-related biological markers over the study duration.
CONDITIONS
Brief Title
AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Ability to understand the study purpose and risks and provide informed consent
- Male or female, at least 18 years old
- Diagnosed with clinically definite or probable ALS by an experienced physician
- Onset of first ALS symptom less than 24 months before study start
- Stable use of riluzole and/or edaravone for at least 30 days before and during the study if treated
- Women of childbearing potential must use birth control during the trial and 60 days after last dose or be non-childbearing
- Female participants or partners must not be pregnant or plan pregnancy during the trial and for 90 days after last dose
- Male participants must avoid sperm donation and use contraception during the trial and for 90 days after last dose
You will not qualify if you...
- Presence of tracheostomy or permanent assisted ventilation
- Forced vital capacity (SVC) less than 65%
- Abnormal liver function tests exceeding specified limits except Gilbert syndrome
- Abnormal kidney function with eGFR below 60 mL/min/1.73m2
- Other lab abnormalities including platelet count or blood clotting times
- Pregnant or breastfeeding women
- Unstable or significant medical conditions other than ALS affecting safety or compliance
- Significant abnormalities in physical, neurological exam, vital signs, or ECG affecting safety
- Unstable psychiatric disease, cognitive impairment, dementia, or substance abuse affecting consent or compliance
- Recent participation in other investigational therapy trials within 30 days prior
- Previous treatment with siRNA, stem cell therapy, ASO, or gene therapy
- Contraindications or disorders affecting lumbar puncture or repeated intrathecal injection
- Prior severe reaction or known allergy to any part of the study drug
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 12 weeks
Participants receive the investigational drug AMX0114 or placebo by intrathecal injection once every 4 weeks for up to 4 doses.
4 visits (in-person) for dosing on Day 1, Day 29, Day 57, and Day 85
Duration - Up to Day 145 (approximately 8 weeks after last dose)
Participants are monitored for safety, tolerability, and pharmacokinetics after completing treatment.
Follow-up visits as scheduled until Day 145
Trial Site Locations
Total: 14 locations
1
University of California, San Diego
La Jolla, California, United States, 92093
Actively Recruiting
2
Georgetown University Hospital Pasquerilla Healthcare Center
Washington D.C., District of Columbia, United States, 20007
Actively Recruiting
3
University of Florida
Gainesville, Florida, United States, 32611
Actively Recruiting
4
Mayo Clinic in Florida
Jacksonville, Florida, United States, 32224
Actively Recruiting
5
Orlando Regional Medical Center, Orlando Health Neuroscience Institute
Orlando, Florida, United States, 32806
Actively Recruiting
6
Massachusetts General Hospital, Healey & AMG Center for ALS
Boston, Massachusetts, United States, 02114
Actively Recruiting
7
Mayo Clinic in Rochester
Rochester, Minnesota, United States, 55905
Actively Recruiting
8
Temple University of the Commonwealth System of Higher Education
Philadelphia, Pennsylvania, United States, 19140
Actively Recruiting
9
Alliance for Multispecialty Research, LLC
Knoxville, Tennessee, United States, 37909
Actively Recruiting
10
Houston Methodist Neurological Institute
Houston, Texas, United States, 77030
Actively Recruiting
11
University of Calgary
Calgary, Alberta, Canada, T2N 4Z6
Actively Recruiting
12
McMaster University
Hamilton, Ontario, Canada, L8N 3Z5
Actively Recruiting
13
London Health Sciences Centre
London, Ontario, Canada, N6G 2M3
Actively Recruiting
14
McGill University Health Centre - Centre for Innovative Medicine
Montreal, Quebec, Canada, H4A 3J1
Actively Recruiting
Research Team
M
Medical Director, Amylyx
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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