An Intermediate Size Expanded Access Protocol of AMX0035 for ALS
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)
1 other identifier
expanded_access
N/A
2 countries
21
Brief Summary
The Expanded Access Program will provide access and assess the safety of AMX0035 for the treatment of people living with ALS.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
21 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 9, 2022
CompletedFirst Posted
Study publicly available on registry
March 18, 2022
CompletedOctober 18, 2022
October 1, 2022
March 9, 2022
October 14, 2022
Conditions
Interventions
Proprietary formulation of sodium phenylbutyrate and taurursodiol
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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);
- +2 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (21)
University of Southern California
Los Angeles, California, 90033, United States
The Kaiser Permanente Medical Group
Oakland, California, 94612, United States
Nova Southeastern University
Fort Lauderdale, Florida, 32608, United States
Holy Cross Health
Fort Lauderdale, Florida, 33308, United States
University of Florida
Gainesville, Florida, 32608, United States
Northwestern University
Chicago, Illinois, 60614, United States
University of Kansas Medical Center
Kansas City, Kansas, 66103, United States
The Sean M. Healey & AMG Center for ALS Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Mayo Clinic
Rochester, Minnesota, 55905, United States
Neurology Associates, P.C. / Somnos Clinical Research
Lincoln, Nebraska, 68510, United States
Columbia University Medical Center
New York, New York, 10032, United States
Duke University Medical Center
Durham, North Carolina, 27705, United States
University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
Thomas Jefferson University Weinberg ALS Center
Philadelphia, Pennsylvania, 19107, United States
Texas Tech University Health Sciences Center El Paso
El Paso, Texas, 79995, United States
Virginia Commonwealth University
Richmond, Virginia, 23298, United States
Swedish Neuroscience Institute
Seattle, Washington, 98122, United States
University of Washington School of Medicine
Seattle, Washington, 98195, United States
Providence St. Luke's Rehabilitation Medical Center
Spokane, Washington, 99202, United States
University of Wisconsin
Madison, Wisconsin, 53705, United States
Hispanic Alliance for Research & Translational Research
San Juan, 00936, Puerto Rico