AMX0114 for ALS (LUMINA Trial)

Recruiting in Palo Alto (17 mi)

Age: 18+

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Recruiting

Sponsor: Amylyx Pharmaceuticals Inc.

Trial Summary

What is the purpose of 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

Adults diagnosed with ALS within the last 24 months can join this trial. They must understand the study, agree to follow its rules, and give informed consent. Women who could get pregnant and men must use birth control during the trial and for some time after. Participants already on certain ALS medications need a stable dose for at least 30 days before starting.

Inclusion Criteria

I have been diagnosed with ALS by an experienced doctor.

I have been on a stable dose of riluzole and/or edaravone for at least 30 days.

My first ALS symptoms started less than 2 years ago.

I am not pregnant and do not plan to become pregnant during the trial and for 90 days after.

Participant Groups

The trial is testing AMX0114 against a placebo in people with ALS. It's a Phase I study focusing on how safe it is, how well it's tolerated, what happens to it in the body (pharmacokinetics), and what effects it has (pharmacodynamics).

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Active Treatment: AMX0114Experimental Treatment1 Intervention

AMX0114 will be administered once every 4 weeks by intrathecal bolus injection for a total of up to 4 doses. Treatment will be administered on Day 1, followed by repeat dosing every 4 weeks at approximately Day 29, Day 57 and Day 85.

Group II: PlaceboPlacebo Group1 Intervention

Placebo drug will be administered once every 4 weeks by intrathecal bolus injection for a total of up to 4 doses. Treatment will be administered on Day 1, followed by repeat dosing every 4 weeks at approximately Day 29, Day 57 and Day 85.