Open Label Extension Study of AMX0035 in Patients With ALS

Amyotrophic Lateral Sclerosis Clinical Trial

— CENTAUR-OLE  

Official title:

Open Label Extension Study of AMX0035 in Patients With ALS

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03488524
• Other study ID #: AMX-3500-OLE
• Status: Completed
• Phase: Phase 2
• Start date: March 29, 2018
• Est. completion date: November 1, 2021

Study information

• Verified date: January 2022
• Source: Amylyx Pharmaceuticals Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Description:

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 95
• Est. completion date: November 1, 2021
• Est. primary completion date: March 1, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• ALS
• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease

Intervention

Drug:
• AMX0035
A combination therapeutic of TUDCA and Sodium Phenylbutyrate

Locations

Country	Name	City	State
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	University of Massachusetts Memorial Medical Center	Worcester	Massachusetts

Sponsors (2)

Lead Sponsor	Collaborator
Amylyx Pharmaceuticals Inc.	Massachusetts General Hospital Neurology Clinical Research Institute

Country where clinical trial is conducted

United States, 

Outcome

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