A phase 1/2a trial of amsulostat, a novel pan-lysyl oxidase inhibitor, in patients with advanced myelofibrosis as an add-on to ruxolitinib treatment for up to 52 weeks
Phase 1/2a trial of amsulostat improved symptom scores and spleen volume in advanced myelofibrosis, indicating potential benefits of prolonged treatment.
Key Points
The trial aims to evaluate the safety and efficacy of amsulostat as an add-on therapy to ruxolitinib in myelofibrosis patients.
Conducted as a Phase 1/2a clinical trial with 16 participants
Patients received amsulostat 200 mg BID for up to 52 weeks alongside ruxolitinib
Assessments included symptom score (MFSAF v4.0), spleen volume, and bone marrow fibrosis
At 24 weeks, 11 patients showed symptom improvements, with 4 achieving a 50% reduction (TSS50)
56% mean reduction in total symptom score at 38 weeks
50% of participants achieved a 25% spleen volume reduction (SVR25) at 38 weeks
Amsulostat was well tolerated with mild treatment-related adverse events noted