Back to resultsOpen Label Extension Study of AMX0035 in Patients With ALS (CENTAUR-OLE)
Primary Purpose
Amyotrophic Lateral Sclerosis, ALS
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
AMX0035
Sponsored by
About this trial
This is an interventional treatment trial for Amyotrophic Lateral Sclerosis
Eligibility Criteria
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.
Sites / Locations
- Massachusetts General Hospital
- University of Massachusetts Memorial Medical Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
AMX0035
Arm Description
AMX0035 twice daily--a combination therapeutic including 3 gram of Phenylbutyrate and 1g TUDCA
Outcomes
Primary Outcome Measures
Quantity of adverse events and serious adverse events observed in the study
Quantity of adverse events and serious adverse events observed in the study
Secondary Outcome Measures
Hospitalizations
Number of Hospitalizations
Rate of Progression on the Amyotrophic Lateral Sclerosis Rating Scale Revised (ALSFRS-R)
Slope of Progression on ALSFRS-R
Rate of Progression on ATLIS Strength Measurement
Rate of progression in strength measurements by ATLIS
Rate of Progression on Slow Vital Capacity
Rate of change in breathing capabilities by slow vital capacity
Gastric Tube Frequency
Number of Gastric Tubes Placed
Permanent Invasive Ventilation
Number of patients of permanent invasive ventilation
Full Information
NCT ID
NCT03488524
First Posted
March 23, 2018
Last Updated
January 5, 2022
Sponsor
Amylyx Pharmaceuticals Inc.
Collaborators
Massachusetts General Hospital Neurology Clinical Research Institute
1. Study Identification
Unique Protocol Identification Number
NCT03488524
Brief Title
Open Label Extension Study of AMX0035 in Patients With ALS
Acronym
CENTAUR-OLE
Official Title
Open Label Extension Study of AMX0035 in Patients With ALS
Study Type
Interventional
2. Study Status
Record Verification Date
January 2022
Overall Recruitment Status
Completed
Study Start Date
March 29, 2018 (Actual)
Primary Completion Date
March 1, 2021 (Actual)
Study Completion Date
November 1, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Amylyx Pharmaceuticals Inc.
Collaborators
Massachusetts General Hospital Neurology Clinical Research Institute
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis, ALS
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Model Description
This an open-label extension study to CENTAUR (AMX-3500)
Masking
None (Open Label)
Allocation
N/A
Enrollment
95 (Actual)
8. Arms, Groups, and Interventions
Arm Title
AMX0035
Arm Type
Experimental
Arm Description
AMX0035 twice daily--a combination therapeutic including 3 gram of Phenylbutyrate and 1g TUDCA
Intervention Type
Drug
Intervention Name(s)
AMX0035
Intervention Description
A combination therapeutic of TUDCA and Sodium Phenylbutyrate
Primary Outcome Measure Information:
Title
Quantity of adverse events and serious adverse events observed in the study
Description
Quantity of adverse events and serious adverse events observed in the study
Time Frame
30 months
Secondary Outcome Measure Information:
Title
Hospitalizations
Description
Number of Hospitalizations
Time Frame
30 months
Title
Rate of Progression on the Amyotrophic Lateral Sclerosis Rating Scale Revised (ALSFRS-R)
Description
Slope of Progression on ALSFRS-R
Time Frame
30 months
Title
Rate of Progression on ATLIS Strength Measurement
Description
Rate of progression in strength measurements by ATLIS
Time Frame
30 months
Title
Rate of Progression on Slow Vital Capacity
Description
Rate of change in breathing capabilities by slow vital capacity
Time Frame
30 months
Title
Gastric Tube Frequency
Description
Number of Gastric Tubes Placed
Time Frame
30 months
Title
Permanent Invasive Ventilation
Description
Number of patients of permanent invasive ventilation
Time Frame
30 months
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Facility Information:
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
University of Massachusetts Memorial Medical Center
City
Worcester
State/Province
Massachusetts
ZIP/Postal Code
01655
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
Undecided
Citations:
PubMed Identifier
35577511
Citation
Paganoni S, Hendrix S, Dickson SP, Knowlton N, Berry JD, Elliott MA, Maiser S, Karam C, Caress JB, Owegi MA, Quick A, Wymer J, Goutman SA, Heitzman D, Heiman-Patterson TD, Jackson C, Quinn C, Rothstein JD, Kasarskis EJ, Katz J, Jenkins L, Ladha SS, Miller TM, Scelsa SN, Vu TH, Fournier C, Johnson KM, Swenson A, Goyal N, Pattee GL, Babu S, Chase M, Dagostino D, Hall M, Kittle G, Eydinov M, Ostrow J, Pothier L, Randall R, Shefner JM, Sherman AV, Tustison E, Vigneswaran P, Yu H, Cohen J, Klee J, Tanzi R, Gilbert W, Yeramian P, Cudkowicz M. Effect of sodium phenylbutyrate/taurursodiol on tracheostomy/ventilation-free survival and hospitalisation in amyotrophic lateral sclerosis: long-term results from the CENTAUR trial. J Neurol Neurosurg Psychiatry. 2022 May 16;93(8):871-5. doi: 10.1136/jnnp-2022-329024. Online ahead of print.
Results Reference
derived
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Open Label Extension Study of AMX0035 in Patients With ALS
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