VEXAS syndrome – European Clinical Trials Information Network

Study of momelotinib in patients with VEXAS syndrome and myelodysplastic syndrome who are dependent on or do not respond to steroid treatment

Wed, 29 Apr 2026 15:07:01 +0000

This clinical trial investigates the use of momelotinib (GSK3070785) in patients with VEXAS syndrome, a rare condition that causes inflammation throughout the body, with or without associated myelodysplastic syndrome, a group of blood disorders where the bone marrow doesn’t produce enough healthy blood cells. The purpose of this study is to determine the most appropriate dose of momelotinib and evaluate how well it works in managing symptoms in patients who have not responded well to steroid treatment.

The study medication will be given as tablets taken by mouth. The treatment period will last up to 48 weeks, during which patients will receive momelotinib at doses up to 300 mg per day. The study consists of two parts: first, a safety phase to find the right dose, followed by a second phase to test how well the medication works.

During the study, doctors will monitor patients’ symptoms and response to treatment, particularly focusing on improvements in VEXAS syndrome symptoms and any changes in blood cell production. They will also track how well patients can reduce their use of steroids while taking the study medication. Throughout the treatment period, patients will have regular check-ups to monitor their health and any side effects.

Study of pacritinib effectiveness and safety compared to placebo in patients with VEXAS syndrome

Wed, 29 Apr 2026 15:04:22 +0000

This clinical trial examines the effectiveness and safety of pacritinib in people with VEXAS syndrome. VEXAS syndrome is a rare condition that causes inflammation in various parts of the body, including the skin, blood vessels, joints, eyes, and lungs. The condition is caused by changes in a gene called UBA1 and typically requires treatment with steroid medications.

The study will test two different doses of pacritinib capsules compared to a placebo. The medication will be taken by mouth. During the first part of the study, participants will not know whether they are receiving the actual medication or the placebo. In the second part, all participants will receive the study medication.

The study will last up to 52 weeks and will measure how well the treatment works by looking at participants’ response to the medication, including improvements in their symptoms and ability to reduce their steroid medication use. The study will also monitor changes in blood test results and track any side effects that may occur during treatment.

Study of Azacitidine Treatment in Patients with VEXAS Syndrome

Wed, 29 Apr 2026 14:29:36 +0000

This clinical trial focuses on VEXAS syndrome, a rare genetic condition that causes inflammation in various parts of the body and can affect blood cell production. The study will test a medication called azacitidine, which will be given as an injection under the skin (subcutaneous injection). This medicine is typically used to treat blood disorders and works by modifying how genes function in the body.

The purpose of this research is to determine if azacitidine can reduce the amount of abnormal genetic material in patients with VEXAS syndrome. The treatment involves receiving azacitidine injections for six treatment cycles. During the study, participants will receive up to 200 milligrams of the medication per day, with a maximum total dose of 10,000 milligrams over a period of up to 10 months.

This is a Phase II study, which means researchers are testing the medication’s effectiveness in a group of patients with VEXAS syndrome. The study will specifically look at how well the treatment works in reducing certain genetic changes associated with the disease. Throughout the treatment period, participants will be monitored for both the medication’s effectiveness and any potential side effects.