An Intermediate Size Expanded Access Protocol of AMX0035 for ALS
- Conditions
- Amyotrophic Lateral Sclerosis
- Registration Number
- NCT05286372
- Lead Sponsor
- Amylyx Pharmaceuticals Inc.
- 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.
Recruitment & Eligibility
- Status
- APPROVED_FOR_MARKETING
- Sex
- All
- Target Recruitment
- Not specified
-
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
Not provided
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (21)
University of Southern California
๐บ๐ธLos Angeles, California, United States
Nova Southeastern University
๐บ๐ธFort Lauderdale, Florida, United States
Northwestern University
๐บ๐ธChicago, Illinois, United States
University of Washington School of Medicine
๐บ๐ธSeattle, Washington, United States
The Sean M. Healey & AMG Center for ALS Massachusetts General Hospital
๐บ๐ธBoston, Massachusetts, United States
Neurology Associates, P.C. / Somnos Clinical Research
๐บ๐ธLincoln, Nebraska, United States
University of Pennsylvania
๐บ๐ธPhiladelphia, Pennsylvania, United States
Duke University Medical Center
๐บ๐ธDurham, North Carolina, United States
The Kaiser Permanente Medical Group
๐บ๐ธOakland, California, United States
University of Florida
๐บ๐ธGainesville, Florida, United States
Holy Cross Health
๐บ๐ธFort Lauderdale, Florida, United States
Mayo Clinic
๐บ๐ธRochester, Minnesota, United States
Columbia University Medical Center
๐บ๐ธNew York, New York, United States
Thomas Jefferson University Weinberg ALS Center
๐บ๐ธPhiladelphia, Pennsylvania, United States
Texas Tech University Health Sciences Center El Paso
๐บ๐ธEl Paso, Texas, United States
Virginia Commonwealth University
๐บ๐ธRichmond, Virginia, United States
Swedish Neuroscience Institute
๐บ๐ธSeattle, Washington, United States
Hispanic Alliance for Research & Translational Research
๐ต๐ทSan Juan, Puerto Rico
University of Wisconsin
๐บ๐ธMadison, Wisconsin, United States
Providence St. Luke's Rehabilitation Medical Center
๐บ๐ธSpokane, Washington, United States
University of Kansas Medical Center
๐บ๐ธKansas City, Kansas, United States