An Intermediate Size Expanded Access Protocol of AMX0035 for ALS

NCT ID: NCT05286372

Last Updated: 2022-10-18

Basic Information

• Recruitment Status: APPROVED_FOR_MARKETING
• Study Classification: EXPANDED_ACCESS

Brief Summary

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

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.

Conditions

Amyotrophic Lateral Sclerosis

Interventions

AMX0035

Proprietary formulation of sodium phenylbutyrate and taurursodiol

Intervention Type: DRUG

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Amylyx Pharmaceuticals Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

University of Southern California

Los Angeles, California, United States

The Kaiser Permanente Medical Group

Oakland, California, United States

Nova Southeastern University

Fort Lauderdale, Florida, United States

Holy Cross Health

Fort Lauderdale, Florida, United States

University of Florida

Gainesville, Florida, United States

Northwestern University

Chicago, Illinois, United States

University of Kansas Medical Center

Kansas City, Kansas, United States

The Sean M. Healey & AMG Center for ALS Massachusetts General Hospital

Boston, Massachusetts, United States

Mayo Clinic

Rochester, Minnesota, United States

Neurology Associates, P.C. / Somnos Clinical Research

Lincoln, Nebraska, United States

Columbia University Medical Center

New York, New York, United States

Duke University Medical Center

Durham, North Carolina, United States

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Thomas Jefferson University Weinberg ALS Center

Philadelphia, Pennsylvania, United States

Texas Tech University Health Sciences Center El Paso

El Paso, Texas, United States

Virginia Commonwealth University

Richmond, Virginia, United States

Swedish Neuroscience Institute

Seattle, Washington, United States

University of Washington School of Medicine

Seattle, Washington, United States

Providence St. Luke's Rehabilitation Medical Center

Spokane, Washington, United States

University of Wisconsin

Madison, Wisconsin, United States

Hispanic Alliance for Research & Translational Research

San Juan, , Puerto Rico

Countries

United States; Puerto Rico

Other Identifiers

A35-006

Identifier Type: -

Identifier Source: org_study_id