Actively Recruiting
AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis
Led by Amylyx Pharmaceuticals Inc. · Updated on 2026-04-14
48
Participants Needed
14
Research Sites
129 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Able to understand the study purpose and risks, willing to comply and provide informed consent
- Male or female, aged 18 years or older
- Diagnosed with clinically definite or probable ALS by an experienced physician
- ALS symptoms started less than 24 months before study start
- If treated with riluzole and/or edaravone, treatment must be stable for at least 30 days before and during the study
- Women of childbearing potential must use acceptable birth control during the study and for 60 days after last dose or be non-childbearing
- Female participants or female partners must not be pregnant or plan pregnancy during the study and for 90 days after last dose
- Male participants must avoid sperm donation and use contraception with female partners during the study and for 90 days after last dose
You will not qualify if you...
- Having a tracheostomy or permanent assisted ventilation
- Forced vital capacity (SVC) less than 65%
- Abnormal liver function with specific enzyme levels above limits unless due to Gilbert syndrome
- Abnormal renal function with eGFR less than 60 mL/min/1.73m2
- Other abnormal lab results including platelet count or blood clotting tests
- Pregnant or breastfeeding women
- Clinically significant unstable medical conditions other than ALS affecting safety or compliance
- Significant abnormalities in physical or neurological exams, vital signs, or ECG affecting safety
- Unstable psychiatric disease, cognitive impairment, dementia, or substance abuse affecting consent or compliance
- Current or recent participation in other investigational therapy trials within 30 days before this study
- Previous treatment with small interfering RNA, stem cell therapy, any ASO, or gene therapy
- Contraindications to lumbar puncture or intrathecal injections
- 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
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
CONTACT
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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