NCT06665165
AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis
Recruiting · Phase 1 · Amylyx Pharmaceuticals Inc. · registry updated 2026-04-14
Inclusion and exclusion lines below are quoted from ClinicalTrials.gov. No match score is shown, because a score needs a person's age, biomarkers, and treatment dates. Confirm the record with the study team.
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).
Inclusion
- 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.
- Male or female, at least 18 years of age.
- Diagnosis of clinically definite or clinically probable ALS, made by a physician who is experienced with management of ALS.
- 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).
- 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.
- 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.
Exclusion
- Presence of tracheostomy or permanent assisted ventilation.
- SVC less than 65%.
- 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.
- Abnormal renal function defined as estimated glomerular filtration rate (eGFR) \< 60 mL/min/1.73m2.
- Other laboratory abnormalities, including abnormalities in platelet count, international normalized ratio, prothrombin time, and activated partial thromboplastin time.
- Pregnant women (confirmed by a pregnancy test within 7 days prior to first dose) or women currently breastfeeding.