AMX0035 consists of a combination of two molecules: tauroursodeoxycholic acid (TUDCA) and sodium phenylbutyrate. Both were previously shown to be safe and well tolerated. The combination of the two drugs is believed to decrease nerve cell damage in people living with ALS.
This phase 3 study is a double-blind, randomized, placebo-controlled study. This means that we compare the effect of AMX0035 against the effect of a placebo. A placebo is a drug without an active ingredient, a ‘fake drug’. The study drug and placebo are supplied in a sachet and must be dissolved in 1 cup of water. Either treatment should be taken orally (ingested) daily for 48 weeks. Participants will be randomly assigned to receive the placebo or AMX0035 treatment. The participant and the research team do not know which treatment the participant is receiving.
During a period of 1 year, participants will visit the research centre approximately 6 times for examinations. They will furthermore be contacted approximately 9 times by telephone or video calling.
Key Eligibility Criteria
The main criteria to participate in this study are:
- You are diagnosed with ALS
- The duration of illness from the first symptoms is no longer than 24 months
- Your lung function is at least 55%, without using a ventilator
- You must not be pregnant or breast-feeding for the entire duration of the study
- If you have or are considering having diaphragm pacing during the study, you cannot participate
- When using Riluzole: a stable dose of ≥30 days prior to participation
Participants can continue their treatment with Riluzole for the entire duration of the study.
Amylyx is looking to enrol 600 participants worldwide. Currently, the study is active in the following European countries: the United Kingdom, Ireland, the Netherlands, Spain, Sweden, Italy, France, Poland, Belgium, Germany and Portugal.