AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis

Conditions

• ALS

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• AMX0114 — DRUG
  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.
• Placebo — OTHER
  Placebo

Study Details

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).

Key Dates

Start date

Apr 7, 2025

Status verified

Apr 2026

Primary completion

Feb 28, 2027

Completion

Oct 31, 2027

Study Design

Enrollment

48 participants (estimated)

Allocation

RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Active Treatment: AMX0114
  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.
• Placebo Comparator: Placebo
  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.

Primary Outcome Measure

Evaluate the safety and tolerability of AMX0114 in adult participants living with ALS [ Time Frame: Day 1 - Day 145 (End of Study) ]

Central Contacts

• Medical Director, Amylyx
  857-320-6200
  [email protected]

Locations (10)

Find similar trials in La Jolla, CA

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