Open Label Extension Study of AMX0035 in Patients With ALS

• Register: ClinicalTrials.gov
• Last refreshed on: 17 January 2022
• Main ID: NCT03488524
• Date of registration: 23/03/2018
• Prospective Registration: Yes
• Primary sponsor: Amylyx Pharmaceuticals Inc.
• Public title: Open Label Extension Study of AMX0035 in Patients With ALS CENTAUR-OLE
• Scientific title: Open Label Extension Study of AMX0035 in Patients With ALS
• Date of first enrolment: March 29, 2018
• Target sample size: 95
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03488524
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Completion of all visits in the randomized, double blind AMX0035 study. Subjects that
receive tracheostomy or PAV during the course of the main study will still be followed
as ITT until the week 24 visit before enrollment in the OLE.

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 open label extension 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 below.

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.



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

ALS

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: AMX0035

Primary Outcome(s)

Quantity of adverse events and serious adverse events observed in the study [Time Frame: 30 months]

Secondary Outcome(s)

Rate of Progression on ATLIS Strength Measurement [Time Frame: 30 months]

Hospitalizations [Time Frame: 30 months]

Rate of Progression on Slow Vital Capacity [Time Frame: 30 months]

Permanent Invasive Ventilation [Time Frame: 30 months]

Gastric Tube Frequency [Time Frame: 30 months]

Rate of Progression on the Amyotrophic Lateral Sclerosis Rating Scale Revised (ALSFRS-R) [Time Frame: 30 months]

Secondary ID(s)

AMX-3500-OLE

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Massachusetts General Hospital Neurology Clinical Research Institute