A Randomized Phase 2/3, Double-blind, Placebo Controlled Adaptive Study Evaluating the Efficacy and Safety of Momelotinib in Participants with VEXAS Syndrome
VEXAS syndrome is a rare inflammatory disorder that affects the blood and various organs. People with VEXAS often experience painful swellings, fevers, skin rashes, and fatigue, and they frequently rely on high doses of steroids that carry difficult side effects. This clinical trial is evaluating whether a tablet called momelotinib is safe and effective for treating VEXAS syndrome. Momelotinib is designed to block signals in the body that drive inflammation and blood issues. In this study, participants are randomly assigned to receive either momelotinib or a dummy pill (placebo). Doctors will measure how well symptoms clear up by week 26 and whether patients can safely reduce their steroid doses. Researchers will also monitor safety, overall well-being, and long-term health for up to three years to see if momelotinib offers lasting benefits.
At a glance
What is this study about?
VEXAS syndrome is a rare condition discovered by scientists in 2020. It causes the body's immune system to attack healthy tissues, leading to fevers, joint pain, skin rashes, and low blood counts. Because it is so new, there are very few approved treatments. Most people have to take high doses of steroids, which can cause significant long-term health problems.
Momelotinib is a targeted medicine taken as a daily tablet. It works by blocking specific proteins known to fuel inflammation and abnormal blood cell production. Researchers hope that by calming this overactive pathway, momelotinib can relieve symptoms and give patients more days without painful flare-ups.
This trial is an integrated Phase 2 and Phase 3 study. This means it first checks that the medicine is safe and working as expected, and then expands to confirm how well it works in a larger group. Half of the participants typically receive momelotinib, while the other half receive a matching dummy tablet (placebo), allowing researchers to prove whether any improvements are truly due to the new medicine.
Taking part in this study helps researchers understand if momelotinib could become one of the first targeted treatment options for people living with VEXAS syndrome.
Key takeaways
- Tests a daily tablet called momelotinib for people with VEXAS syndrome.
- Aims to reduce inflammation, symptom flare-ups, and the need for steroids.
- Open to adults aged 18 and older diagnosed with VEXAS syndrome.
- Compares the active medicine against a dummy tablet (placebo).
- Involves regular clinic visits and health tracking for up to three years.
Who may be eligible?
This trial is open to adults aged 18 and over who have been diagnosed with VEXAS syndrome. Men and women can both take part, though VEXAS is far more common in men.
To join, participants must have active symptoms or ongoing inflammation that requires treatment, typically including steroid therapy. Doctors will check that your heart, kidneys, and liver are working well enough for you to take part safely.
You may not be eligible if you have certain active infections, other severe medical conditions, or if you cannot swallow tablets. Your study doctor will review your full medical history and current medications to ensure the trial is safe for you.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- I am aged 18 or older.
- I have a confirmed diagnosis of VEXAS syndrome.
- I currently have active symptoms or require steroid treatment.
- I am able to swallow tablets every day.
- I can attend regular hospital clinic visits for blood tests and health checks.
What does participation involve?
If you take part, you will be randomly assigned to take either momelotinib tablets or matching dummy tablets by mouth each day. Neither you nor your doctor will get to choose which treatment you receive.
You will visit the hospital clinic regularly for health checks, especially during the first 26 weeks. These visits will include blood and urine tests, physical examinations, and questions about your daily symptoms and quality of life. You will also track your daily steroid use to see if your dose can be lowered.
After the initial 26-week assessment, your health will continue to be monitored up to 52 weeks and then periodically for up to three years. This long-term follow-up helps doctors understand how the treatment affects your health over time.
Potential risks and benefits
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Common questions
What is VEXAS syndrome?
VEXAS syndrome is a rare genetic condition that causes the immune system to create severe inflammation, leading to fevers, skin rashes, joint pain, and blood problems.
Will I definitely receive momelotinib?
No. This is a randomised study, meaning a computer randomly chooses whether you receive momelotinib or a dummy tablet (placebo).
Can I stay on my current steroid medication?
Yes. Most participants will continue taking their steroid medicine, and the study team will see if your dose can be safely reduced over time.
How often will I need to visit the clinic?
Visits are more frequent during the first 26 weeks for safety checks and blood tests, becoming less frequent during the long-term follow-up period.
What happens if my symptoms get worse?
Your medical team will monitor you closely. If your condition flares up or gets worse, the doctors will step in to adjust your care and keep you safe.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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