A Phase 3, Randomized, Double-Blind, Active-Comparator-Controlled Study of Bomedemstat Versus Hydroxyurea in Patients with Essential Thrombocythemia Naïve to Cytoreductive Therapy
Abstract
Background and Significance: Essential thrombocythemia (ET) is a myeloproliferative neoplasm characterized by thrombocytosis, increased risk of thrombosis, and the potential to develop myelofibrosis (MF) and acute myeloid leukemia (AML). ET is driven by mutations in JAK2, CALR, and MPL. Clinical management is focused on reducing the incidence of thrombotic and bleeding events. Currently available treatments can normalize platelet counts (PC) but are not effective in all patients. There remains an unmet need for novel therapies that can alter the natural history of ET. Bomedemstat (MK-3543) is an irreversible inhibitor of lysine-specific demethylase 1, an enzyme that regulates hematopoietic stem and progenitor cell proliferation and maturation, including differentiation to megakaryocytes. Bomedemstat reduced PC, white cell counts (WCC), and serum levels of inflammatory cytokines in preclinical models of myeloproliferative neoplasms. In a phase 2 clinical study of patients with ET, bomedemstat improved symptoms, durably reduced PC and WCC, and reduced mutation burden. This randomized, double-blind, active-comparator-controlled, phase 3 study (NCT06456346) will investigate the efficacy and safety of bomedemstat versus hydroxyurea in patients with high-risk ET who are naive to cytoreductive therapy.
Study Design and Methods: Eligible patients are aged ≥18 years and have cytoreductive therapy naive ET with an indication for cytoreductive therapy, a bone marrow fibrosis score of 0…
How strong is the evidence?
Evidence Hub reads this as a randomized trial, done in people. Its evidence level is 2 of 5 (High · randomized trial). Randomized trials of a reasonable size are the strongest single-study design. Reported size: 300 participants or records.
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