Momelotinib in VEXAS Syndrome

To be considered for this study, you must be at least 18 years old and have a confirmed diagnosis of VEXAS syndrome with a specific genetic change (UBA1 mutation). Your VEXAS symptoms should be active and currently require daily steroid treatment of at least 10mg. You might be eligible if your current treatments, including steroids, aren't fully controlling your symptoms, or if you find yourself needing higher and higher doses of steroids.

There are also some health checks you'll need to pass. For instance, your liver and kidney functions must be within certain healthy limits. If you are taking other medications that affect your immune system, you might need a short break from them before starting momelotinib. Certain growth factor treatments for blood conditions are also not allowed shortly before the study. We will check your overall health to make sure you are well enough to participate.

If you are a woman who could become pregnant, you'll need to have a negative pregnancy test and agree to use effective contraception throughout the study and for 12 weeks afterwards. If you are a man, you’ll also need to use a condom if you have sexual activity with a woman who could become pregnant. These steps are important to prevent pregnancy during the study because the effects of momelotinib on a developing baby are not known.

If you join this study, you will first go through a screening period to check if you're a good fit. During this time, your doctor will carefully decide the right dose of steroids you need daily to manage your VEXAS symptoms. Once you start treatment, you will take momelotinib every day at a dose determined by the study, along with your prescribed steroids.

Your health will be regularly monitored with hospital visits. Doctors will evaluate your VEXAS symptoms after 4, 12, 24, and 48 weeks. If you also have MDS, those features will be checked at 12 and 24 weeks. You will continue taking momelotinib until your VEXAS symptoms worsen, you no longer respond to the treatment, or if the doctors decide, in your best interest, to stop it. If the treatment isn't helping after 24 weeks at the optimal dose, it will be stopped. The study involves regular check-ups and medication, potentially for many months, but you can choose to leave the study at any time.