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.



France