Open Label Extension Study of AMX0035 in Patients With ALS

Purpose

This extension study, in which all participants received active treatment (AMX0035), was designed to assess the longer-term safety and therapeutic potential of AMX0035 for participants who have completed the Main Study (AMX3500, also known as CENTAUR).

Conditions

  • Amyotrophic Lateral Sclerosis
  • ALS

Eligibility

Eligible Ages
All ages
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  1. Completion of all visits in the randomized, double blind AMX3500 study. Participants who received tracheostomy or permanent assisted ventilation (PAV) during the course of the main study could enroll in the OLE if they completed all visits in the main study. 2. Must enroll in the OLE within 28 days of the Week 24 visit of the main study. 3. Signed informed consent to enter the OLE phase.

Exclusion Criteria

  1. Discontinued study drug prematurely in the double-blind phase of the study for reasons other than tracheostomy or PAV. 2. Exposure to or anticipated requirement for any disallowed medication listed in the protocol. 3. Any ongoing adverse events that in the opinion of the Site Investigator are clear contraindications to the study drug. 4. Unstable cardiac or other life-threatening disease emergent during the randomized, double blind study 5. Any major medical condition that in the opinion of the Site Investigator would interfere with the study and place the subject at increased risk.

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
N/A
Intervention Model
Single Group Assignment
Intervention Model Description
This is an open-label extension study to CENTAUR (AMX3500)
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
AMX0035 		AMX0035 twice daily--a combination of Sodium Phenylbutyrate (3g) and Taurursodiol (1g)
  • Drug: AMX0035
    Combination therapy of PB and TURSO
    Other names:
    • Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO, also known as Ursodoxicoltaurine or Tauroursodeoxycholic Acid [TUDCA])

More Details

Status
Completed
Sponsor
Amylyx Pharmaceuticals Inc.

Study Contact

Detailed Description

The Centaur Open Label Extension Study (CENTAUR-OLE) is an extension study for patients with ALS who participated in the CENTAUR study (Study AMX3500). During the OLE, all participants received active treatment (AMX0035), and the investigators, evaluators, and participants remained blinded to the randomized treatment assigned at the beginning of the double-blind main study. The study was designed to assess the longer term safety and therapeutic potential of AMX0035.