AMX0035 for ALS
(Phoenix Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called AMX0035 for people with ALS, a disease that affects nerve cells in the brain and spinal cord, causing muscle weakness. Researchers aim to determine if AMX0035 is safe and effective for treating ALS symptoms. Participants will receive either AMX0035 or a placebo (a substance with no active treatment) to compare effects. Those diagnosed with ALS within the last 24 months and able to follow trial procedures might be suitable for this study. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to help potentially bring a new treatment to market.
Do I need to stop my current medications to join the trial?
The trial does not specify if you need to stop taking your current medications, but if you are on riluzole or edaravone, you must have been on a stable dose for at least 14 days or a full treatment cycle, respectively. Check with the trial team for any specific medications that might be prohibited.
Is there any evidence suggesting that AMX0035 is likely to be safe for humans?
Research has shown that AMX0035 has undergone safety testing in people with ALS (Amyotrophic Lateral Sclerosis). Previous studies found that AMX0035, a combination of phenylbutyrate and ursodoxicoltaurine, was generally well-tolerated. The primary goal was to confirm its safety for long-term use. Most participants managed the treatment without serious issues, and while some experienced side effects, these were mostly mild. If someone cannot tolerate the treatment, adjustments such as changing the dosage can be made. Overall, the safety data so far suggests AMX0035 could be a safe option for people with ALS.12345
Why do researchers think this study treatment might be promising for ALS?
Researchers are excited about AMX0035 for ALS because it combines two compounds, sodium phenylbutyrate and taurursodiol, which may protect nerve cells from damage. Unlike standard ALS treatments like riluzole or edaravone, which mainly aim to slow disease progression, AMX0035 targets both neuronal inflammation and cell death pathways, potentially offering a more comprehensive approach. This dual-action mechanism could mean more effective management of ALS symptoms and improved quality of life for patients.
What evidence suggests that AMX0035 might be an effective treatment for ALS?
Research has shown that AMX0035, one of the treatments studied in this trial, may help treat ALS, a progressive nerve disease. In earlier studies, participants taking AMX0035 experienced a slower decline in physical abilities compared to those taking a placebo, which contains no active medicine. The CENTAUR study found that AMX0035 was safe and helped slow the disease's progression. AMX0035 consists of two ingredients, sodium phenylbutyrate and taurursodiol, which work together to protect nerve cells from damage. These findings suggest that AMX0035 could be a beneficial option for managing ALS symptoms.13467
Who Is on the Research Team?
Amylyx Study Director
Principal Investigator
Amylyx Pharmaceuticals
Are You a Good Fit for This Trial?
This trial is for adults diagnosed with ALS within the last 2 years who can consent and follow study procedures. They must use birth control during the study and not plan to conceive. Excluded are pregnant or breastfeeding women, those with severe medical conditions, on salt restriction, enrolled in other trials, using prohibited meds, or have specific health issues like low lung capacity or poor kidney function.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive AMX0035 or placebo for 48 weeks to evaluate safety and efficacy in ALS treatment
Follow-up
Participants are monitored for safety and effectiveness after treatment
Long-term follow-up
Long-term survival will be monitored by assessing all-cause mortality
What Are the Treatments Tested in This Trial?
Interventions
- AMX0035
- Placebo
Trial Overview
The Phoenix Trial is testing AMX0035 against a placebo to see if it's safe and effective for treating ALS. This Phase III trial randomly assigns participants to either receive AMX0035 or a placebo without them knowing which one they're getting (double-blind).
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Placebo Group
Placebo administered by mouth or via feeding tube for 48 weeks: once daily for first 3 weeks and then twice daily for remainder of study if participant tolerating
Placebo administered by mouth or via feeding tube for 48 weeks: once daily for first 3 weeks and then twice daily for remainder of study if participant tolerating
AMX0035 is already approved in United States, Canada for the following indications:
- Amyotrophic Lateral Sclerosis
- Amyotrophic Lateral Sclerosis
Find a Clinic Near You
Who Is Running the Clinical Trial?
Amylyx Pharmaceuticals Inc.
Lead Sponsor
Citations
Amylyx Pharmaceuticals Announces Topline Results From ...
The Phase 3 PHOENIX study enrolled 664 adults living with ALS. Participants were randomized three-to-two to receive either AMX0035 or placebo.
Clinical Review - Sodium phenylbutyrate-ursodoxicoltaurine ...
The primary safety outcome of the CENTAUR study was to confirm the safety and tolerability of PB-TURSO while the primary efficacy outcome was the rate of change ...
Efficacy of Sodium Phenylbutyrate and Ursodoxicoltaurine ...
Variants in the actin polymerization protein profilin 1 (PFN1) have been implicated as a genetic cause of amyotrophic lateral sclerosis ...
AMX0035 and Oral Edaravone for Amyotrophic Lateral ...
Model results therefore represent average findings across patients and should not be presumed to represent the clinical or cost outcomes for any.
216660Orig1s000 CLINICAL AND STATISTICAL REVIEW(S)
The proposed indication for AMX0035 is for the treatment of amyotrophic lateral sclerosis (ALS). ... effectiveness of sodium phenylbutyrate/taurursodiol (AMX0035) ...
6.
ec.europa.eu
ec.europa.eu/health/documents/community-register/2024/20240105160995/anx_160995_en.pdfAlbrioza, INN-Sodium phenylbutyrate/Ursodoxicoltaurine
AMX0035 is a fixed-dose combination consisting of phenylbutyrate (PB) and ursodoxioltaurine (TURSO). It is hypothesized to reduce neuronal cell death and ...
Sodium Phenylbutyrate and Ursodoxicoltaurine (Albrioza)
CADTH recommends that Albrioza be reimbursed by public drug plans for the treatment of patients with amyotrophic lateral sclerosis (ALS) if certain conditions
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