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Quick Info
Status
Not Yet Recruiting
Phase
-
Trial Type
Expanded Access
Treatment Type
Drug Trials
Randomization
None (EAP)
Enrollment
Start Date
Contact Information
    Contact information unknown.
Locations
Puerto Rico, Other
Hispanic Alliance for Research & Translational Research, San Juan, 00936, Puerto Rico
United States, Illinois
Northwestern University, Chicago, IL, 60614, United States
Contact: Emma Schmidt   312-503-4362   emma.schmidt@northwestern.edu
United States, Kansas
University of Kansas Medical Center, Kansas City, KS, 66103, United States
Contact: Katie Lillig   913-945-9932   Kjennens2@Kumc.edu
United States, Massachusetts
The Sean M. Healey & AMG Center for ALS Massachusetts General Hospital, Boston, MA, 02114, United States
Contact: Max Higgins   617-643-2522   mphiggins@mgh.harvard.edu
United States, Minnesota
Mayo Clinic, Rochester, MN, 55905, United States
United States, Nebraska
Neurology Associates, P.C. / Somnos Clinical Research, Lincoln, NE, 68510, United States
Contact: 402-770-7403
United States, North Carolina
Duke University Medical Center, Durham, NC, 27705, United States
Contact: Sarah Parker   919-681-4191   ALSresearch@duke.edu
United States, Pennsylvania
Thomas Jefferson University Weinberg ALS Center, Philadelphia, PA, 19107, United States
Contact: Hristelina Ilieva, MD, PhD
United States, Virginia
Virginia Commonwealth University, Richmond, VA, 23298, United States
Contact: LaVon Smith   804-360-4669   lavon.smith@vcuhealth.org
United States, Washington
Swedish Neuroscience Institute, Seattle, WA, 98122, United States
Contact: Janae Rahiman   206-320-4120
Providence St. Luke's Rehabilitation Medical Center, Spokane, WA, 99202, United States
Contact: Ann Cooper, LPN, CCRC   509-474-4320   ann.cooper@providence.org
United States, Wisconsin
University of Wisconsin, Madison, WI, 53705, United States
Trial Goal
Tests to Expect
Open Label
Yes
Update Notes
No significant updates
6/22/2022
Sites added
6/13/2022
Sites added
6/6/2022
Site updates
5/23/2022
Sites added
5/16/2022
New site added
5/10/2022
Sites added
4/29/2022
Contact update
3/22/2022

Other Information

Purpose
The Expanded Access Program will provide access and assess the safety of AMX0035 for the treatment of people living with ALS.
Eligibility
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
Details
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.
Collaborator(s)
  • Amylyx Pharmaceuticals Inc.
Trial Protocol as Published on Clinicaltrials.gov
NCT05286372 (First Published: 3/9/2022)