AMX0114 for ALS
This study is testing AMX0114, an investigational drug, in adults with Amyotrophic Lateral Sclerosis (ALS). AMX0114 is an antisense oligonucleotide (ASO) that works by reducing the amount of a protein called calpain-2 in the body. Researchers want to see how safe and tolerable AMX0114 is for people with ALS. They will also look at how the drug affects certain markers in the body related to ALS. You may be able to join if you are at least 18 years old and have a diagnosis of definite or probable ALS. This study plans to enroll 48 participants, but its current recruitment status is unclear.
- Study design
- This is a placebo-controlled Phase I study, meaning some participants will receive AMX0114 and others will receive a placebo (an inactive substance). It aims to enroll 48 participants.
- What's involved
- AMX0114 is given by intrathecal injection (an injection into the spinal canal in the lower back). Researchers will monitor for side effects and conduct various medical assessments.
- Compensation
- Not stated in the trial record.
- Follow-up
- The safety and tolerability of AMX0114 will be evaluated from Day 1 to Day 145 (End of Study).
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis
At a glance
Conditions
NCT06665165
Where you'd take part
This study runs at 14 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Alliance for Multispecialty Research, LLC
Knoxville, Tennesseestudy coordinator listed
Recruiting
Georgetown University Hospital Pasquerilla Healthcare Center
Washington D.C., District of Columbiastudy coordinator listed
Recruiting
Houston Methodist Neurological Institute
Houston, Texasstudy coordinator listed
Recruiting
London Health Sciences Centre
London, Ontario, Canadastudy coordinator listed
Recruiting
Massachusetts General Hospital, Healey & AMG Center for ALS
Boston, Massachusettsstudy coordinator listed
Recruiting
Mayo Clinic in Florida
Jacksonville, Floridastudy coordinator listed
Recruiting
Mayo Clinic in Rochester
Rochester, Minnesotastudy coordinator listed
Recruiting
McGill University Health Centre - Centre for Innovative Medicine
Montreal, Quebec, Canadastudy coordinator listed
Recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Study leadership
- Medical Director, Amylyx · STUDY_DIRECTOR · Medical Monitor
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
What this trial measures
- Evaluate the safety and tolerability of AMX0114 in adult participants living with ALSDay 1 - Day 145 (End of Study)
Incidence of adverse events (AEs), serious adverse events (SAEs) and dose limiting toxicities (DLTs). Incidence of abnormalities in clinical laboratory assessments, vital signs, physical and neurological examinations, and electrocardiograms (ECGs).