Open Label Extension Study of AMX0035 in Patients With ALS

Study Overview
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Brief Summary
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This study will provide extended access to patients and assess longer-term outcomes on patients who have completed the Centaur study.

Detailed Description
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The Centaur Open Label Extension Study (CENTAUR-OLE) is designed to provide longer term access to AMX0035 for patients with ALS who participated in the CENTAUR study. The study will assess longer term safety and therapeutic potential of AMX0035.

Official Title
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Open Label Extension Study of AMX0035 in Patients With ALS

Conditions
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ALS, Amyotrophic Lateral Sclerosis

Intervention / Treatment
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* Drug: AMX0035


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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. Must enroll in the OLE within 28 days of the Week 24 visit of the main study. Signed informed consent to enter the open label extension phase. Exclusion Criteria: Discontinued study drug prematurely in the double-blind phase of the study for reasons other than tracheostomy or PAV. Exposure to or anticipated requirement for any disallowed medication listed below. Any ongoing adverse events that in the opinion of the Site Investigator are clear contraindications to the study drug. Unstable cardiac or other life-threatening disease emergent during the randomized, double blind study Any major medical condition that in the opinion of the Site Investigator would interfere with the study and place the subject at increased risk.
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: This an open-label extension study to CENTAUR (AMX-3500)
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: AMX0035<br>AMX0035 twice daily--a combination therapeutic including 3 gram of Phenylbutyrate and 1g TUDCA | Drug: AMX0035<br>* A combination therapeutic of TUDCA and Sodium Phenylbutyrate<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Quantity of adverse events and serious adverse events observed in the study | Quantity of adverse events and serious adverse events observed in the study | 30 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Hospitalizations | Number of Hospitalizations | 30 months | 
| Rate of Progression on the Amyotrophic Lateral Sclerosis Rating Scale Revised (ALSFRS-R) | Slope of Progression on ALSFRS-R | 30 months | 
| Rate of Progression on ATLIS Strength Measurement | Rate of progression in strength measurements by ATLIS | 30 months | 
| Rate of Progression on Slow Vital Capacity | Rate of change in breathing capabilities by slow vital capacity | 30 months | 
| Gastric Tube Frequency | Number of Gastric Tubes Placed | 30 months | 
| Permanent Invasive Ventilation | Number of patients of permanent invasive ventilation | 30 months |