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A study to investigate the safety and efficacy of AMX0035 in patients with Amyotrophic Lateral Sclerosis
EU CTIS ID: 2023-508511-23-00
What this study is testing
To assess the impact of AMX0035 treatment compared to placebo on disease progression over 48 weeks in adult patients with ALS.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Male or female, at least 18 years of age.
- Men must not plan to father a child or to provide sperm for donation for the duration of the trial and 3 months after the last dose of study drug.
- Diagnosis of ALS (clinically definite or clinically probable), made by a physician who is experienced with management of ALS, as defined by the World Federation of Neurology revised El Escorial criteria.
- Time since onset of first symptom of ALS should be <24 months.
- If the participant is to be treated with riluzole and/or edaravone during the course of the trial, then treatment with riluzole and/or edaravone was, at the time of the baseline visit, previously started and maintained at a stable regimen for at least 14 days for riluzole and/or for a full treatment cycle for edaravone.
- Capable of providing informed consent.
You likely can't join if
- Presence of tracheostomy or PAV.
- Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the participant to provide informed consent, according to Investigator judgment.
- Clinically significant unstable medical condition (other than ALS) that would pose a risk to the participant if he/she were to participate in the trial, according to Investigator judgment
- Previous treatment for ALS with cellular therapies or gene therapies
- Currently enrolled on another trial involving use of an investigational therapy
- Previous treatment with PB or taurursodiol within 30 days from Screening
See the full eligibility criteria
Who can join
- Male or female, at least 18 years of age.
- Men must not plan to father a child or to provide sperm for donation for the duration of the trial and 3 months after the last dose of study drug.
- Diagnosis of ALS (clinically definite or clinically probable), made by a physician who is experienced with management of ALS, as defined by the World Federation of Neurology revised El Escorial criteria.
- Time since onset of first symptom of ALS should be <24 months.
- If the participant is to be treated with riluzole and/or edaravone during the course of the trial, then treatment with riluzole and/or edaravone was, at the time of the baseline visit, previously started and maintained at a stable regimen for at least 14 days for riluzole and/or for a full treatment cycle for edaravone.
- Capable of providing informed consent.
- Capable and willing to follow trial procedures including visits to the trial clinic, remote visits, and status reporting requirements.
- Women of childbearing potential (WOCBP; 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 trial and 3 months after the last dose of study drug.
- Women must not be pregnant or planning to become pregnant for the duration of the trial and 3 months after last dose of study drug.
- Men must agree to practice contraception for the duration of the trial and for at least 3 months after last dose of study drug
What rules you out
- Presence of tracheostomy or PAV.
- Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the participant to provide informed consent, according to Investigator judgment.
- Clinically significant unstable medical condition (other than ALS) that would pose a risk to the participant if he/she were to participate in the trial, according to Investigator judgment
- Previous treatment for ALS with cellular therapies or gene therapies
- Currently enrolled on another trial involving use of an investigational therapy
- Previous treatment with PB or taurursodiol within 30 days from Screening
- Implantation of Diaphragm Pacing System
- Currently or previously treated within the last 30 days or planned exposure to any prohibited medications.
- Slow Vital Capacity less than 55%.
- History of known allergy to Phenylbutyrate or bile salts.
- Abnormal liver function
- Renal insufficiency as defined by estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73m2 (obtained within 12 weeks from first dose).
- Pregnant women (confirmed by a pregnancy test within 7 days of first dose) or women currently breastfeeding.
- Current severe biliary disease which may result in the Investigator medical judgment in biliary obstruction including for example active cholecystitis, primary biliary cirrhosis, sclerosing cholangitis, gallbladder cancer, 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
The study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Kingdom; United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.