An Intermediate Size Expanded Access Protocol of AMX0035 for ALS

October 14, 2022 updated by: Amylyx Pharmaceuticals Inc.

An Intermediate Size, Expanded Access Protocol to Provide AMX0035, a Fixed Combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO), for the Treatment of Adult Patients With Amyotrophic Lateral Sclerosis (ALS)

The Expanded Access Program will provide access and assess the safety of AMX0035 for the treatment of people living with ALS.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Approved for marketing

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

AMX0035 is a combination therapy designed to reduce neuronal death through blockade of key cellular death pathways originating in the endoplasmic reticulum (ER) and mitochondria. This Expanded Access Program is designed to provide expanded access to AMX0035 for the treatment of people living with ALS and assess safety in diverse populations/stages of ALS over 48 weeks.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Expanded Access

Expanded Access Type

Expanded Access Type

Describes the category of expanded access under U.S. Food and Drug Administration (FDA) regulations. There are three types of expanded access:
  • Individual Patients: Allows a single patient, with a serious disease or condition who cannot participate in a clinical trial, access to a drug or biological product that has not been approved by the FDA. This category also includes access in an emergency situation.
  • Intermediate-size Population: Allows more than one patient (but generally fewer patients than through a Treatment IND/Protocol) access to a drug or biological product that has not been approved by the FDA. This type of expanded access is used when multiple patients with the same disease or condition seek access to a specific drug or biological product that has not been approved by the FDA.
  • Treatment IND/Protocol: Allows a large, widespread population access to a drug or biological product that has not been approved by the FDA. This type of expanded access can only be provided if the product is already being developed for marketing for the same use as the expanded access use.
  • Intermediate-size Population

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Puerto Rico
      • San Juan, Puerto Rico, 00936
        • Hispanic Alliance for Research & Translational Research
  • United States
    • California
      • Los Angeles, California, United States, 90033
        • University of Southern California
      • Oakland, California, United States, 94612
        • The Kaiser Permanente Medical Group
    • Florida
      • Fort Lauderdale, Florida, United States, 33308
        • Holy Cross Health
      • Fort Lauderdale, Florida, United States, 32608
        • Nova Southeastern University
      • Gainesville, Florida, United States, 32608
        • University of Florida
    • Illinois
      • Chicago, Illinois, United States, 60614
        • Northwestern University
    • Kansas
      • Kansas City, Kansas, United States, 66103
        • University of Kansas Medical Center
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • The Sean M. Healey & AMG Center for ALS Massachusetts General Hospital
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Mayo Clinic
    • Nebraska
      • Lincoln, Nebraska, United States, 68510
        • Neurology Associates, P.C. / Somnos Clinical Research
    • New York
      • New York, New York, United States, 10032
        • Columbia University Medical Center
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • Duke University Medical Center
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • University of Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19107
        • Thomas Jefferson University Weinberg ALS Center
    • Texas
      • El Paso, Texas, United States, 79995
        • Texas Tech University Health Sciences Center El Paso
    • Virginia
      • Richmond, Virginia, United States, 23298
        • Virginia Commonwealth University
    • Washington
      • Seattle, Washington, United States, 98195
        • University of Washington School of Medicine
      • Seattle, Washington, United States, 98122
        • Swedish Neuroscience Institute
      • Spokane, Washington, United States, 99202
        • Providence St. Luke's Rehabilitation Medical Center
    • Wisconsin
      • Madison, Wisconsin, United States, 53705
        • University of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Male or female, at least 18 years of age (inclusive);
  • Diagnosis of ALS made by a physician experienced with the management of ALS;
  • >36 months from symptom onset defined as first weakness
  • Capable of providing informed consent;
  • Capable of and willing to follow program procedures.
  • Participants who have established care with a physician experienced in treating patients with ALS involved in the program and will maintain this clinical care throughout the duration of their time in the program.

  • Women of childbearing potential (e.g., not post-menopausal for at least one year or surgically sterile) must agree to use adequate birth control for the duration of the program and 3 months after last dose of AMX0035;

    • Women must not be planning to become pregnant for the duration of the program and 3 months after last dose of study drug

  • Men must agree to practice contraception for the duration of the program and for at least 3 months after last dose of program drug;

    • Men must not plan to father a child or provide sperm

Exclusion Criteria

  • Currently enrolled in a therapeutic study involving the use of an investigational therapy;

  • Dependence on invasive mechanical ventilation, defined as being unable to lay supine without it, unable to sleep without it, or continuous daytime use; presence of tracheostomy at Screening;

    • No current need for tracheostomy or PAV (defined as more than 22 hours daily of mechanical ventilation for more than one week (7 days) or based on the site investigator's judgment; no need anticipated for the next 12 weeks
  • In the judgment of the Investigator, the participant's expected survival is less than 6 months
  • History of known allergy to the following: PB, bile salts, excipient/constituents of the formulation;
  • Abnormal liver function defined as AST and/or ALT >3 times the upper limit of the normal (obtained within 12 weeks from first dose);
  • Renal insufficiency as defined by eGFR <60 mL/min/1.73m2 (obtained within 12 weeks from first dose);
  • Pregnant women or women currently breastfeeding;
  • Current severe biliary disease which may result in the investigator medical judgement in biliary obstruction including for example active cholecystitis, primary biliary cirrhosis, sclerosing cholangitis, gallbladder cancer, gallbladder polyps, gangrene of the gallbladder, abscess of the gallbladder;
  • History of Class III/IV heart failure (per New York Heart Association - NYHA);
  • Participant under severe salt restriction where the added salt intake due to treatment would put the participant at risk, in the Site Investigator clinical judgment;
  • Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the participant to provide informed c consent, according to Site Investigator judgment;
  • Clinically significant unstable medical condition (other than ALS) that would pose a risk to the participant if they were to participate in the program, according to Site Investigator judgment;
  • Treatment, current or within 90 days from screening with any cell therapies or gene therapies;
  • Implantation of Diaphragm Pacing System (DPS);
  • Anything that, in the opinion of the Site Investigator, would place the participant at increased risk or preclude the participant's full compliance with or completion of the program;
  • Current or planned exposure to any prohibited medications listed in Section 6.8.1 of the protocol

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Amylyx Pharmaceuticals Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
March 9, 2022

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 9, 2022

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 18, 2022

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
October 18, 2022

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 14, 2022

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
October 1, 2022

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • A35-006

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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