Shorespan-007: Phase 3 study of bomedemstat versus hydroxyurea in essential thrombocythemia naive to cytoreductive therapy
Abstract
TPS6587
Background:
Lysine-specific demethylase 1 (LSD1) is an enzyme that regulates hematopoietic stem and progenitor cell proliferation and maturation. Bomedemstat (MK-3543) is an LSD1 inhibitor shown to have manageable safety and improve symptoms, durably reduce platelet and white blood cell (WBC) count, and reduce mutation burden in patients with essential thrombocythemia (ET) in a phase 2 study. Here, we describe the methodology of the randomized, double-blind, phase 3 Shorespan-007 study (NCT06456346), which has been designed to evaluate the efficacy and safety of bomedemstat compared with hydroxyurea in participants with ET naive to cytoreductive therapy.
Methods:
Key eligibility criteria include patients aged ≥18 years with an ET diagnosis per WHO diagnostic criteria for myeloproliferative neoplasms, an indication for cytoreductive therapy, no prior cytoreductive therapy, a bone marrow fibrosis score of 0 or 1, a platelet count of >450 × 10
9
/L, and an absolute neutrophil count of ≥0.75 × 10
9
/L. Key exclusion criteria include a documented increased risk of bleeding or an active infection necessitating systemic therapy. Approximately 300 participants will be enrolled. Participants will be randomly assigned 1:1 to bomedemstat at a starting dose of 50 mg/day by mouth titrated to a target platelet count of ≥150× 10
9
/L to ≤350 × 10
9
/L or hydroxyurea at a starting dose of 500 mg/day by mouth titrated per the approved product labeling. The primary end point is…
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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