VERIFY: A randomized controlled phase 3 study of the hepcidin mimetic rusfertide (PTG-300) in patients with polycythemia vera (PV).

作者：Aniket Bankar, Kristen Pettit, Joseph J. Shatzel, Abdulraheem Yacoub, Naveen Pemmaraju, Harinder Gill, Antonín Hluší, Alessandro Lucchesi, Samah Alimam, Jiří Mayer, Francesca Palandri, Daniel Sasca, Katharina Modelska, Suneel Gupta, Ifode Ajari, Sarita Khanna, Arturo Molina, Andrew Kuykendall · 发表于：Journal of Clinical Oncology · 年份：2024 · DOI：10.1200/jco.2024.42.16_suppl.tps6592 · 被引用次数：3 · 研究领域：Myeloproliferative Neoplasms: Diagnosis and Treatment、Hemoglobinopathies and Related Disorders、Cytokine Signaling Pathways and Interactions

TPS6592 Background: PV is a myeloproliferative neoplasm characterized by overproduction of red blood cells and increased risk of thrombosis. Patients may require frequent therapeutic phlebotomies (TP) alone or in combination with cytoreductive therapy to maintain hematocrit (HCT) <45%. Hepcidin regulates iron homeostasis but is downregulated in PV, increasing iron availability for erythropoiesis, which complicates TP optimization. In a phase 2 study (REVIVE, NCT04057040), rusfertide led to rapid, sustained, and durable HCT control with over 90% of patients achieving TP independence in part 2. 1 It was well tolerated; majority (77.1%) of treatment-emergent adverse events (TEAEs) had a maximum grade of 2. There were no Grade 4 or 5 TEAEs. The phase 3 study VERIFY (NCT05210790) aims to confirm efficacy and safety of rusfertide in patients with PV. Methods: VERIFY is a multicenter, global, randomized trial comparing efficacy and safety of rusfertide (starting dose: 20 mg subcutaneously once weekly) vs. placebo when added to ongoing therapy for PV. Patients with PV who require frequent TP with or without concurrent cytoreductive therapy to control HCT are included in this 3-part study: Part 1a: 1:1 randomized, double-blind, placebo-controlled, add-on parallel-group period lasting 32 weeks (Week 0-32); Part 1b: open-label treatment phase with cross-over for previous placebo-treated patients. During this phase, all patients who completed Part 1a successfully will receive rusferti...