AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis
ConditionALS
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorAmylyx Pharmaceuticals Inc.
About this trial
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 criteria
Qualifiers
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).
Disqualifiers
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.
Trial design
Treatments tested in this trial
- AMX0114
- Placebo
Treatment groups
48 Participants
are divided into 2 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov