Study of Momelotinib in Patients With VEXAS Syndrome With or Without Associated Myelodysplastic Syndrome Phase II with Safety Run-In

What this Trial Is About

Researchers are evaluating the efficacy and safety of momelotinib in patients with VEXAS syndrome, a rare inflammatory condition often accompanied by myelodysplastic syndrome (MDS). This phase II trial includes a safety run-in phase to determine the maximum tolerated dose of momelotinib, followed by a single-arm prospective phase where patients receive the drug at this dose. The study addresses the need for effective treatment in patients with symptomatic VEXAS requiring immunosuppressive therapy. The trial begins with a safety run-in phase testing three fixed doses of momelotinib (150 mg, 200 mg, and 300 mg once daily) using small patient cohorts to find the highest dose that is well tolerated. After this, up to 39 patients will receive momelotinib continuously at the determined maximum tolerated dose during the phase II portion until disease progression or loss of response. All participants will start with glucocorticoids (prednisone or equivalent) at baseline, and the dose is adjusted during screening to control symptoms. Treatment may be stopped after 24 weeks without response or if adverse effects occur. Participants will be monitored closely with assessments of VEXAS symptoms at 4, 12, 24, and 48 weeks, and evaluations of MDS features at 12 and 24 weeks. Other measures include pharmacokinetics, adverse events, hematological response, steroid dose reduction, survival, and molecular markers. The study involves regular visits to assess response, safety, and disease progression, lasting up to 48 weeks of treatment with follow-up as needed.

Momelotinib in VEXAS Syndrome