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GSK启动eltrombopag骨髓增生异常综合症III期SUPPORT研究

来源:生物谷 2014-07-01 07:42

2014年7月1日讯 /生物谷BIOON/ --葛兰素史克(GSK)近日宣布,启动III期SUPPORT(TRC112121)研究。该研究在中级-1、中级-2或高危骨髓增生异常综合症(MDS)患者中开展,将调查eltrombopag(Promacta/Revolade,艾曲波帕)+阿扎胞苷(azacitidine,当前标准护理)组合疗法相对于安慰剂+阿扎胞苷组合疗法的疗效。研究中将评估前4个治疗周期内无需血小板输注的患者比例。

骨髓增生异常综合症(MDS)是起源于造血干细胞的一组异质性髓系克隆性疾病,特点是髓系细胞分化及发育异常,表现为无效造血、血细胞减少、造血功能衰竭,高风险向急性髓系白血病(AML)转化。高达45%的患者会经历一定时期MDS后会转化成AML。MDS治疗主要解决两大问题:骨髓衰竭及并发症、AML转化。就患者群体而言,MDS患者自然病程和预后的差异性很大,治疗宜个体化。

今年3月,GSK向FDA提交了Promacta的补充新药申请(sNDA),寻求批准该药用于对免疫抑制疗法(IST)响应不足的重型再生障碍性贫血(SAA)患者血细胞减少症(cytopenia)的治疗。此前,FDA已授予Promacta治疗SAA的突破性疗法认定。重型再生障碍性贫血(SAA)是一种罕见性疾病,患者骨髓无法制造足够的新的血细胞。目前,还没有药物获批用于对免疫抑制疗法(IST)无响应的SAA患者的治疗。对初始IST响应不足的SAA患者群体,约40%的患者会在疾病确诊5年内,死于感染或出血。

关于Eltrombopag(艾曲波帕):

目前,Eltrombopag已获全球100多个国家批准,用于慢性免疫(特发性)血小板减少性紫癜(ITP)患者血小板减少症(thrombocytopenia)的治疗,同时已获43个国家批准用于慢性丙型肝炎(CHC)患者血小板减少症的治疗,以便启动并维持以干扰素为基础的肝病标准疗法。

eltrombopag能够与促血小板生成素(TPO)受体相互作用,从而增加血小板的生成,该药在美国的商品名为Promacta,在欧洲及其他国家和地区的商品名为Revolade。(生物谷Bioon.com)

英文原文:GSK announces the start of a phase III study with eltrombopag in patients with myelodysplastic syndromes

GlaxoSmithKline plc (LSE/NYSE: GSK) today announced the start of a Phase III study, SUPPORT (TRC112121), to evaluate the platelet supportive care effects of eltrombopag (Promacta™/Revolade™) in combination with azacitidine (the current standard of care) versus placebo in combination with azacitidine in intermediate-1, intermediate-2 or high risk patients with myelodysplastic syndromes (MDS). The global study will assess the proportion of patients who are platelet transfusion free during the first four cycles of treatment.

MDS is a type of cancer in which the bone marrow does not make enough healthy blood cells and there are abnormal (blast) cells in the blood and/or bone marrow.[i] The disease usually manifests itself with 1 or more cytopenias, or reductions in the number of blood cells, and patients typically present with complications related to anaemia (fatigue), neutropenia (infections), or thrombocytopenia (bleeding).[ii]  MDS may evolve into acute myeloid leukaemia (AML) in up to 45 percent of patients.[iii]

About the SUPPORT study

SUPPORT (StUdy of eltromboPag in myelodysPlastic SyndrOmes Receiving azaciTidine) is a Phase III, randomised, double-blind, placebo-controlled, multi-centre study of eltrombopag or placebo in combination with azacitidine in subjects with MDS. It is estimated that 350 patients across 30 countries and 156 study sites with a baseline platelet count of < 75Gi/L, and intermediate-1, intermediate-2 or high risk MDS (by International Prognostic Scoring System) will be enrolled in the study. Eligible patients will be randomised to receive either eltrombopag (200 mg once daily [100 mg for East Asians]) plus azacitidine (75 mg/m2 subcutaneously once daily for 7 days) every 28 days, for at least 6 cycles, or placebo plus azacitidine. Dose modifications of eltrombopag or placebo will be permitted to ensure that patient platelet counts remain at a safe and effective level.

The primary efficacy endpoint will determine the platelet supportive care effects of eltrombopag in combination with azacitidine versus placebo in combination with azacitidine by comparing the proportion of subjects receiving eltrombopag plus azacitidine who are platelet transfusion free during the first four cycles of azacitidine, versus those treated with placebo plus azacitidine. Secondary objectives will compare the following between treatment arms: overall survival, disease response, haematologic improvement, platelet and red blood cell transfusions, adverse events (≥ Grade 3), safety and tolerability, health related quality of life and medical resource utilization.

About eltrombopag

Eltrombopag—marketed as Promacta™ in the U.S. and as Revolade™ in Europe and other countries across the world—works by interacting with the TPO receptor leading to increased platelet production. Eltrombopag is not approved or licensed anywhere in the world for use in patients with myelodysplastic syndromes.

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