Amyotrophic Lateral SclerosisNCT05286372
An Intermediate Size Expanded Access Protocol of AMX0035 for ALS
A study testing AMX0035 in people with Amyotrophic Lateral Sclerosis, run by Amylyx Pharmaceuticals Inc. at 21 sites in 2 countries.
Trial summary
The Expanded Access Program will provide access and assess the safety of AMX0035 for the treatment of people living with ALS. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- AMX0035
- How many people
- Not given
- Where
- 21 sitesin 2 countries
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Adult, Older adult
Who may be able to join
- 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.
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- 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
Who cannot join
- 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);
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- 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
Trial locations and contacts
21 sites in 2 countries.
21 sites in 2 countries
United States20 sites
The Sean M. Healey & AMG Center for ALS Massachusetts General Hospital
Boston, Massachusetts, 02114
Northwestern University
Chicago, Illinois, 60614
Duke University Medical Center
Durham, North Carolina, 27705
Texas Tech University Health Sciences Center El Paso
El Paso, Texas, 79995
Nova Southeastern University
Fort Lauderdale, Florida, 32608
Holy Cross Health
Fort Lauderdale, Florida, 33308
University of Florida
Gainesville, Florida, 32608
University of Kansas Medical Center
Kansas City, Kansas, 66103
Neurology Associates, P.C. / Somnos Clinical Research
Lincoln, Nebraska, 68510
University of Southern California
Los Angeles, California, 90033
University of Wisconsin
Madison, Wisconsin, 53705
Columbia University Medical Center
New York, New York, 10032
The Kaiser Permanente Medical Group
Oakland, California, 94612
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Thomas Jefferson University Weinberg ALS Center
Philadelphia, Pennsylvania, 19107
Virginia Commonwealth University
Richmond, Virginia, 23298
Mayo Clinic
Rochester, Minnesota, 55905
Swedish Neuroscience Institute
Seattle, Washington, 98122
University of Washington School of Medicine
Seattle, Washington, 98195
Providence St. Luke's Rehabilitation Medical Center
Spokane, Washington, 99202
Puerto Rico1 site
Hispanic Alliance for Research & Translational Research
San Juan, 00936
About this study
Official title: 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.
Read the 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
- Being tested
- AMX0035
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AMX0035Drug
Proprietary formulation of sodium phenylbutyrate and taurursodiol
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Mar 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Amylyx Pharmaceuticals Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05286372
- Sponsor's protocol ID
- A35-006
- Status last verified
- Oct 2022
- First submitted
- 9 Mar 2022
- Last updated
- 18 Oct 2022
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT05286372
- Acronym / sponsor ID
- A35-006
- Conditions
- Amyotrophic Lateral Sclerosis
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Amylyx Pharmaceuticals Inc.Industry (pharma, biotech, devices)
- First posted
- 18 Mar 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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.
Key exclusion criteria (17 listed)
- 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;
- 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);
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AMX0035Proprietary formulation of sodium phenylbutyrate and taurursodiol | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Oct 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
21 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing AMX0035 in people with Amyotrophic Lateral Sclerosis, run by Amylyx Pharmaceuticals Inc. at 21 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 18 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 21 sites in United States, Puerto Rico. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Oct 2022.