A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS)
This study is looking into a new medication called pacritinib for people with VEXAS syndrome. VEXAS is a rare inflammatory condition. The main goal is to find out if pacritinib is safe and effective at preventing flare-ups and helping people reduce their steroid medication. Participants will be aged 18 or older and currently taking steroids for their VEXAS. Some will get pacritinib, others a dummy pill (placebo). It's a 'double-blind' study, meaning neither you nor your doctor will know which you're getting at first. After this initial period, all participants will have the chance to receive pacritinib. The study hopes to offer a new treatment option for this challenging condition.
At a glance
What is this study about?
This study is testing a new medicine called pacritinib for a condition known as VEXAS syndrome. VEXAS is a rare inflammatory disease that can affect different parts of the body, causing symptoms like skin rashes, joint pain, and other issues. People with VEXAS often need to take steroid medications, like prednisone, to control their symptoms.
The main aim of this study is to see if pacritinib is both safe and effective. Researchers want to find out if taking pacritinib can help prevent VEXAS flare-ups and allow people to reduce their steroid dose. For a while, some participants will receive pacritinib and others will receive a dummy pill (called a placebo). This helps researchers accurately compare the drug's effects. After this initial period, everyone in the study will have the opportunity to receive pacritinib.
This is a 'Phase 2' study, which means it's still in the earlier stages for this specific condition, but pacritinib has been studied for other conditions. The study will last for quite some time, potentially over two years for some participants, to thoroughly understand how the medication works long-term. By taking part, you could help doctors learn more about VEXAS and potentially help develop new treatments for others with the condition.
Key takeaways
- Tests a new medication, pacritinib, for VEXAS syndrome.
- Aims to reduce VEXAS flare-ups and steroid use.
- Involves a 'double-blind' comparison with a placebo for the first 24 weeks.
- All participants will eventually have the opportunity to receive pacritinib.
- Closely monitors your health and condition throughout the study.
- Could help advance understanding and treatment of VEXAS syndrome.
Who may be eligible?
To join this study, you need to be at least 18 years old and have a confirmed diagnosis of VEXAS syndrome, meaning a specific gene change (mutation) has been found. You must also have experienced VEXAS-related inflammation in certain body parts, like skin, joints, or blood vessels, within the past six months.
Another key requirement is that you must currently be taking steroids, such as prednisone or prednisolone, at a stable dose between 15 and 45 mg per day for at least four weeks before joining. Your general health should be good enough to participate, and doctors will check various blood tests to make sure your liver, kidneys, and other important systems are working well. They will also look at your heart rhythm.
There will be other specific health checks to ensure the study is safe for you. If you have any questions about whether you might be eligible, it's best to speak with your doctor or the study team.
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.
- Are you 18 years old or older?
- Do you have a confirmed diagnosis of VEXAS syndrome with a specific gene change?
- Have you had VEXAS-related inflammation affecting your body in the last 6 months?
- Are you currently taking a stable dose of steroids (15-45 mg/day) for VEXAS?
- Is your general health good enough to participate?
- Are you able to attend regular clinic visits?
What does participation involve?
If you join this study, you will be assigned by chance (like flipping a coin) to one of three groups. Two groups will receive different doses of pacritinib, and one group will receive a placebo (a dummy pill with no active medication). Neither you nor your study doctor will know which treatment you are getting during the first part of the study, which lasts up to 24 weeks. This is called 'double-blind'.
Throughout this time, you will have regular visits to the clinic for check-ups, blood tests, and to discuss how you're feeling. After the first 24 weeks, or sooner if needed, all participants will switch to receiving pacritinib without knowing the dose for up to another 48 weeks. If you are doing well and your doctor believes you are benefiting, you might be able to continue receiving pacritinib for an additional year. If you stop the study treatment at any point, there will be a follow-up period of 30 days to check on your health. The total study duration, including all follow-up, could be over two years.
Potential risks and benefits
Locations (39)
- Mayo Clinic - ScottsdaleVerified postcodeScottsdale, United States· Recruiting
- Dana Farber Cancer InstituteVerified postcodeBoston, United States· Recruiting
- Mayo Clinic - RochesterVerified postcodeRochester, United States· Recruiting
- NYU Langone HealthVerified postcodeNew York, United States· Recruiting
- Cleveland Clinic - ClevelandVerified postcodeCleveland, United States· Recruiting
- The James Cancer Hospital and Solove Research InstituteVerified postcodeColumbus, United States· Recruiting
- UT MD Anderson Cancer CenterVerified postcodeHouston, United States· Recruiting
- University of Utah HealthcareVerified postcodeSalt Lake City, United States· Recruiting
- Fred Hutchinson Cancer CenterVerified postcodeSeattle, United States· Recruiting
- Vancouver Coastal Health Research InstituteVerified postcodeVancouver, Canada· Recruiting
- Queen Elizabeth II Health Sciences CenterVerified postcodeHalifax, Canada· Recruiting
- Princess Margaret Cancer CentreVerified postcodeToronto, Canada· Recruiting
Common questions
What is VEXAS syndrome?
VEXAS syndrome is a rare inflammatory condition that can cause various symptoms affecting different parts of the body, often requiring steroid treatment.
What is Pacritinib?
Pacritinib is the new investigational medication being tested in this study to see if it can help manage VEXAS syndrome.
What is a placebo?
A placebo is a 'dummy pill' that looks exactly like the study medication but contains no active drug. It helps researchers compare the real drug's effects accurately.
How long will the study last?
The study involves a treatment period of up to 24 weeks, followed by another period of up to 48 weeks, and potentially an additional year. The total duration could be over two years for some participants.
Will I know if I'm getting the drug or the placebo?
For the first part of the study (up to 24 weeks), neither you nor your doctor will know if you are receiving pacritinib or the placebo. After this, all participants will have the chance to receive pacritinib.
How to find out more
Study Physician
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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