Open Label Extension Study of AMX0035 in Patients With ALS

Phase 2

Completed

• Conditions: Amyotrophic Lateral Sclerosis; ALS
• Interventions: Drug: AMX0035

• Registration Number: NCT03488524
• Lead Sponsor: Amylyx Pharmaceuticals Inc.

Brief Summary

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

Detailed 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.

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations
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Related News

Study Timeline

• Study First Submitted: 2018-03-23
• Study Start: 2018-03-29
• Study First Submitted QC: 2018-04-03
• Study First Posted: 2018-04-05
• Primary Completion: 2021-03-01
• Study Completion: 2021-11-01
• Status Verified: 2022-01
• Last Update Submitted: 2022-01-05
• Last Update Posted: 2022-01-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 95

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.

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
AMX0035	AMX0035	AMX0035 twice daily--a combination therapeutic including 3 gram of Phenylbutyrate and 1g TUDCA

Primary Outcome Measures

Name	Time	Method
Quantity of adverse events and serious adverse events observed in the study	30 months	Quantity of adverse events and serious adverse events observed in the study

Secondary Outcome Measures

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

Trial Locations

Locations (2)

Massachusetts General Hospital; 🇺🇸 Boston, Massachusetts, United States

University of Massachusetts Memorial Medical Center; 🇺🇸 Worcester, Massachusetts, United States