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Open Label Extension Study of AMX0035 in Patients With ALS (CENTAUR-OLE)

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ClinicalTrials.gov Identifier: NCT03488524
Recruitment Status : Recruiting
First Posted : April 5, 2018
Last Update Posted : April 13, 2018
Sponsor:
Collaborator:
Massachusetts General Hospital Neurology Clinical Research Institute
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis ALS Drug: AMX0035 Phase 2

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.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 132 participants
Intervention Model: Single Group Assignment
Intervention Model Description: This an open-label extension study to CENTAUR (AMX-3500)
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open Label Extension Study of AMX0035 in Patients With ALS
Actual Study Start Date : March 29, 2018
Estimated Primary Completion Date : January 1, 2020
Estimated Study Completion Date : January 1, 2020


Arm Intervention/treatment
Experimental: AMX0035
AMX0035 twice daily--a combination therapeutic including 3 gram of Phenylbutyrate and 1g TUDCA
Drug: AMX0035
A combination therapeutic of TUDCA and Sodium Phenylbutyrate




Primary Outcome Measures :
  1. Quantity of adverse events and serious adverse events observed in the study [ Time Frame: 12 months ]
    Quantity of adverse events and serious adverse events observed in the study


Secondary Outcome Measures :
  1. Hospitalizations [ Time Frame: 12 months ]
    Number of Hospitalizations

  2. Rate of Progression on the Amyotrophic Lateral Sclerosis Rating Scale Revised (ALSFRS-R) [ Time Frame: 12 months ]
    Slope of Progression on ALSFRS-R

  3. Rate of Progression on ATLIS Strength Measurement [ Time Frame: 12 months ]
    Rate of progression in strength measurements by ATLIS

  4. Rate of Progression on Slow Vital Capacity [ Time Frame: 12 months ]
    Rate of change in breathing capabilities by slow vital capacity

  5. Gastric Tube Frequency [ Time Frame: 12 months ]
    Number of Gastric Tubes Placed

  6. Permanent Invasive Ventilation [ Time Frame: 12 months ]
    Number of patients of permanent invasive ventilation



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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03488524


Contacts
Contact: Emily Engel 617-724-7210 Emily.Engel@mgh.harvard.edu

Locations
United States, Massachusetts
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
Contact: Aileen Shaughnessy, MA    617-724-9196    ANSHAUGHNESSY@mgh.harvard.edu   
Principal Investigator: James Berry, MD         
University of Massachusetts Memorial Medical Center Recruiting
Worcester, Massachusetts, United States, 01655
Contact: Diane McKenna-Yasek, RN, BSN    508-856-4697    diane.mckenna-yasek@umassmed.edu   
Principal Investigator: Margaret Owegi, DO         
Sponsors and Collaborators
Amylyx Pharmaceuticals Inc.
Massachusetts General Hospital Neurology Clinical Research Institute

Responsible Party: Amylyx Pharmaceuticals Inc.
ClinicalTrials.gov Identifier: NCT03488524     History of Changes
Other Study ID Numbers: AMX-3500-OLE
First Posted: April 5, 2018    Key Record Dates
Last Update Posted: April 13, 2018
Last Verified: April 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases