Dazucorilant in Patients With Amyotrophic Lateral Sclerosis

This study is looking for adults aged 18 and over who have been diagnosed with ALS, whether it runs in their family or appeared without a clear family history. To be considered, your disease progression needs to be within a certain range as estimated by doctors during screening. If you are already taking other approved ALS medications like riluzole or edaravone, you must be on a steady dose before joining the study.

There are certain situations that would prevent you from taking part. For example, if you have other serious neurological conditions not related to ALS, or have difficulty swallowing capsules, you won't be able to join. Your kidney function, blood platelet levels, and certain infections (like HIV or hepatitis B/C) will also be checked, as these could affect your eligibility. Women who are pregnant, planning to become pregnant, or breastfeeding cannot participate.

Also, if you're using breathing support like a non-invasive ventilator, a breathing tube, or oxygen supplementation, you wouldn't be able to join this study. For specific parts of the study, there might be additional rules, such as restrictions on a particular gene mutation (SOD1) or a specific combination therapy (sodium phenylbutyrate and taurursodiol).

If you decide to take part, the study involves regular visits to the clinic for check-ups and assessments. You'll be given the study medication as capsules to take daily. In the first part of the study, you'd take either dazucorilant or a placebo for 24 weeks, without knowing which one it is. If you complete this, you might have the option to continue taking dazucorilant for up to 132 weeks (about 2 and a half years) in an 'open-label' part, where everyone receives the active drug. If you don't enter the open-label part, you'll still have a follow-up period of 132 weeks. In the second part of the study, you'll start with a lower dose of dazucorilant, and this dose will be gradually increased over time until you reach the target dose, provided it's well-tolerated. This part can also lead into a 52-week open-label extension. The total duration of your participation would vary depending on which part of the study you join and whether you continue into the extension phases.