5613260 A PHASE -- IIA-IIB, OPEN LABEL, SINGLE CENTER TRIAL TO STUDY SAFETY, TOLERABILITY AND EFFICACY OF MEMANTINE AS SUPPORTIVE LONG-TERM TREATMENT OF SICKLE CELL PATIENTS: TRIAL DESIGN AND ENROLLMENT

5613260 一项 IIA-IIB 期、开放标签、单中心试验,旨在研究美金刚作为镰状细胞病患者长期支持治疗的安全性、耐受性和有效性:试验设计和入组

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Abstract

Selinexor is an orally bioavailable selective inhibitor of nuclear export compound that inhibits exportin-1 (XPO1), a novel therapeutic target that is overexpressed in multiple myeloma (MM) and is responsible for the transport of ∼220 nuclear proteins to the cytoplasm, including tumour suppressor proteins. Inhibition of this process has demonstrated substantial antimyeloma activity in preclinical studies, both alone and in combination with established MM therapeutics. Based on a clinical trial programme encompassing multiple combination regimens, selinexor-based therapy has been approved for the treatment of relapsed/refractory MM (RRMM), with selinexor-dexamethasone approved in the later-relapse setting for penta-refractory patients and selinexor-bortezomib-dexamethasone approved for patients who have received ≥1 prior therapy. Here, we provide a comprehensive review of the clinical data on selinexor-based regimens, including recent updates from the 2022 American Society of Hematology annual meeting, and summarise ongoing studies of this novel targeted agent in newly diagnosed MM and RRMM.

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