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AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis

NCT06665165 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 48

Last updated 2026-04-14

No results posted yet for this study

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).

Conditions

Interventions

DRUG

AMX0114

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.

OTHER

Placebo

Placebo

Sponsors & Collaborators

  • Amylyx Pharmaceuticals Inc.

    lead INDUSTRY

Principal Investigators

  • Medical Director, Amylyx · Medical Monitor

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-04-07
Primary Completion
2027-02-28
Completion
2027-10-31
FDA Drug
Yes

Countries

  • United States
  • Canada

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06665165 on ClinicalTrials.gov