Phase 1, Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose Study to Evaluate Safety, Tolerability, PK and PD of Antisense Oligonucleotide AMX0114 Administered to Adult Participants With Amyotrophic Lateral Sclerosis
Amylyx Pharmaceuticals Inc.
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).
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 ele…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. 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. 2. Male or female, at least 18 years of age. 3. Diagnosis of clinically definite or clinically probable ALS, made by a physician who is experienced with management of ALS. 4. 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). 5. If the participant is to be…
Interventions
- DrugAMX0114
Antisense oligonucleotides (ASOs) are a type of medicine that treats diseases by intercepting the mRNA messages sent within the cell, resulting in fewer specific proteins being made. AMX0114 is an ASO that targets the mRNA messenger that instructs the body to create a protein called calpain-2. Calpain-2 has been linked to the degeneration and death of neurons in many neurological diseases, including people living with sporadic ALS. AMX0114 is designed to reduce the levels of calpain-2, with the goal of slowing down the process that leads to neuron injury and death.
- OtherPlacebo
Placebo
Locations (14)
- University of California, San DiegoLa Jolla, California
- Georgetown University Hospital Pasquerilla Healthcare CenterWashington D.C., District of Columbia
- University of FloridaGainesville, Florida
- Mayo Clinic in FloridaJacksonville, Florida
- Orlando Regional Medical Center, Orlando Health Neuroscience InstituteOrlando, Florida
- Massachusetts General Hospital, Healey & AMG Center for ALSBoston, Massachusetts