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NCT06665165 · ClinicalTrials.gov registry record · Phase 1

AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis

A Phase 1 study of ALS, sponsored by Amylyx Pharmaceuticals.

Recruiting
Registry status
Phase 1
Development phase
48
Enrollment target
14
Study locations

NCT06665165: Recruiting Phase 1 study of ALS, sponsored by Amylyx Pharmaceuticals.

NCT06665165 is a Phase 1 study of ALS that is actively recruiting participants, run by Amylyx Pharmaceuticals. The registered enrollment target is 48 participants, below the 1,034-participant average among 19 other ALS trials with a reported enrollment target (95% lower). The trial reports 14 study locations across 11 states. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT06665165, a Phase 1 study of ALS, is actively recruiting participants, sponsored by Amylyx Pharmaceuticals.

RECRUITING
Registry status
Phase 1
Development phase
48 participants
Enrollment target
14
Study locations

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).

Primary Outcome

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).

Conditions Studied

ALS

Interventions

  • OTHER Placebo
  • DRUG AMX0114

Study Locations (14)

Florida

  • University of Florida - Gainesville
  • Mayo Clinic in Florida - Jacksonville
  • Orlando Regional Medical Center, Orlando Health Neuroscience Institute - Orlando

Ontario

  • McMaster University - Hamilton
  • London Health Sciences Centre - London

California

  • University of California, San Diego - La Jolla

District of Columbia

  • Georgetown University Hospital Pasquerilla Healthcare Center - Washington D.C.

Massachusetts

  • Massachusetts General Hospital, Healey & AMG Center for ALS - Boston

Minnesota

  • Mayo Clinic in Rochester - Rochester

Pennsylvania

  • Temple University of the Commonwealth System of Higher Education - Philadelphia

Tennessee

  • Alliance for Multispecialty Research, LLC - Knoxville

Trial Details

FieldValue
Enrollment Target 48 participants
Start Date 2025-04-07
Est. Completion 2027-10
Phase Phase 1
Amylyx Pharmaceuticals

9 total trials

What NCT06665165 shows while recruiting

NCT06665165 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 48 participants, a relatively small participant target, below the 1,034-participant average among 19 other ALS trials with a reported enrollment target (95% lower).

The record links to 1 condition, with ALS appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.

NCT06665165 lists 14 locations in 11 states (Florida, Ontario, California).

Frequently Asked Questions

What is clinical trial NCT06665165 about?

NCT06665165 is a clinical study titled "AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis". 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).

What is the current status of trial NCT06665165?

This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 48 participants. The study started on 2025-04-07. Estimated completion is 2027-10.

What conditions does trial NCT06665165 study?

This clinical trial studies the following conditions: ALS.

What interventions are being tested in trial NCT06665165?

The interventions under investigation include: Placebo (OTHER), AMX0114 (DRUG).

Who is sponsoring clinical trial NCT06665165?

This trial is sponsored by Amylyx Pharmaceuticals, which has 9 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT06665165 being conducted?

This trial has 14 study locations across California, District of Columbia, Florida, Massachusetts, Minnesota. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Learn More About Clinical Trials

Similar trials for ALS

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Where NCT06665165's enrollment target sits among peer trials

48 12th of 19 higher than 8 of 19 other ALS trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other ALS trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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More recruiting context for NCT06665165

Source: ClinicalTrials.gov NCT06665165, the US trial registry maintained by the National Library of Medicine. NCT06665165 (small enrollment · multi site footprint · recruiting) retrieved and formatted by PlainTrial, see methodology.