本帖最后由 菡萏尘雾 于 2012-11-28 11:52 编辑
消息来源http://www.cytokinetics.com/press_releases/release/pr_1351490133
CYTOKINETICS ANNOUNCES OPENING OF BENEFIT-ALS, A PHASE IIB CLINICAL TRIAL OF TIRASEMTIV (CK-2017357) IN PATIENTS WITH AMYOTROPHIC LATERAL SCLEROSIS
CYTOKINETICS公司宣布开启BENEFIT-ALS项目，即TIRASEMTIV (CK-2017357)针对ALS的IIB（II期B阶段）临床实验
BENEFIT-ALS Will Evaluate Longer Term Effects of Novel Skeletal Muscle Activator and Represents a Key Step Forward Towards Potential Registration
BENEFIT-ALS项目将评估这一新型骨骼肌活化剂的中长期疗效，这意味着向潜在的获得批准迈出了关键的一步
South San Francisco, CA - October 29, 2012
南旧金山，加州，2012年10月29日
Cytokinetics, Incorporated (Nasdaq: CYTK) announced that the company has opened BENEFIT-ALS (Blinded Evaluation of Neuromusclar Effects and Functional Improvement with Tirasemtiv in ALS), formerly known as CY 4026, to enrollment. BENEFIT-ALS is a Phase IIb, multi-national, double-blind, randomized, placebo-controlled, clinical trial designed to evaluate the safety, tolerability and potential efficacy of tirasemtiv (formerly CK-2017357) in patients with amyotrophic lateral sclerosis (ALS).
Cytokinetics公司（纳斯达克：CYTK）宣布已经开启BENEFIT-ALS项目，（针对ALS的Tirasemtiv 实验，盲法评估在神经肌肉和功能改善方面的效果）。也就是之前的CY4026项目，招募参试人员。BENEFIT-ALS是II期b阶段、多国家、双盲、随机、安慰剂对照的临床实验。旨在评估tirasemtiv (之前称为 CK-2017357)针对ALS的安全性、耐受性和潜在的疗效。
Tirasemtiv selectively activates the fast skeletal muscle troponin complex by increasing its sensitivity to calcium, which increases skeletal muscle force in response to neuronal input and delays the onset and reduces the degree of muscle fatigue. Tirasemtiv is the lead drug candidate that has emerged from the company’s skeletal muscle contractility program.
Tirasemtiv 可以通过增加其钙离子敏感性而选择性激活快速肌纤维肌钙蛋白复合体，这将增加骨骼肌对神经输入信号的响应力度，从而延缓肌肉萎缩，减轻肌肉萎缩的程度。Tirasemtiv 在是公司骨骼肌收缩计划中处于领先地位的候选药物。
“Patients who suffer from this devastating disease are in critical need of a novel therapy that addresses the functional deficits that limit their activities of daily living,” stated Jeremy M. Shefner, MD, PhD, Professor and Chair of the Department of Neurology at the Upstate Medical University at the State University of New York and Principal Investigator of BENEFIT-ALS. “If successful, this novel mechanism therapy could improve the lives of many patients living with ALS.”
“深受这种毁灭性疾病折磨的病人，迫切需要一种新的疗法，以解决限制他们行动和日常生活的功能缺失” Jeremy M. Shefner（医学博士，纽约州立医科大学神经病学系主任、教授，BENEFIT-ALS项目首席研究员）说，“一旦成功，这种新机制的疗法可以提高很多ALS病人的生存质量”
BENEFIT-ALS is designed to enroll approximately 400 patients who will first complete one week of treatment with open-label tirasemtiv at 125 mg twice daily. Following completion of the open-label period, patients will be randomized to receive 12 weeks of double-blind treatment with twice-daily oral ascending doses of tirasemtiv beginning at 125 mg twice daily and increasing weekly up to 250 mg twice daily or a dummy dose titration with placebo. Clinical assessments will take place monthly during the course of treatment; patients will also participate in follow-up evaluations one and four weeks after their final dose.
BENEFIT-ALS项目设计为招募大约400名病人，他们将首先接受一周的非盲治疗，125毫克tirasemtiv 每日两次。非盲实验之后，病人将随机接受12周的双盲治疗，每日两次口服，剂量逐步增加。剂量从125毫克开始，随后增加到250毫克。或者是虚拟剂量的安慰剂治疗。治疗过程中每月会进行一次临床评估。病人服药之后一周和四周，还将接受后续的评估。
The primary efficacy analysis of BENEFIT-ALS will compare the mean change from baseline in the ALS Functional Rating Scale in its revised form (ALSFRS-R) on tirasemtiv versus placebo. Secondary endpoints will include Maximum Voluntary Ventilation (MVV) and other measures of respiratory and skeletal muscle function. Patients taking riluzole at the time of enrollment and who are randomized to receive tirasemtiv will receive riluzole at a reduced dose of 50 mg daily. Cytokinetics plans to conduct this trial at over 70 sites across the United States, Canada, and several European countries.
BENEFIT-ALS在疗效分析方面，主要是比较tirasemtiv与安慰剂组ALS功能评定量表修订形式从基线开始的平均差别。次级的评定点将包括最大自主通气量(MVV)和其他反映呼吸肌和骨骼肌功能的测量指标。招募时在服用利鲁唑的病人和随机接受tirasemtiv 治疗的病人，服用利鲁唑，但减量的每日50毫克。Cytokinetics公司计划在美国、加拿大和好几个欧洲国家超过70个地区进行该实验。
“We are pleased to open BENEFIT-ALS to enrollment to further evaluate tirasemtiv in patients with ALS. The 12 weeks of double-blind treatment in this trial will be the longest time that any ALS patients have been treated with tirasemtiv; consequently, BENEFIT-ALS will be a critical step forward in our assessment of the potential for this treatment as a chronic therapy,” stated Andrew A. Wolff, MD, FACC, Cytokinetics’ Senior Vice President of Clinical Research and Development and Chief Medical Officer. “Based on feedback from health regulatory authorities in the United States, Canada and the European Union, we believe that BENEFIT-ALS may support the potential registration of tirasemtiv for the treatment of ALS.”
“我们很高兴能开启BENEFIT-ALS招募参试人员以进一步评估tirasemtiv对ALS的疗效。12周的双盲实验是所有ALS病人接受tirasemtiv以来最长的。因此，这是我们评定这种药物作为慢性疗法的关键一步。”Andrew A. Wolff（医学博士，美国心脏病学会会员，Cytokinetics公司临床研发高级副总裁，首席医疗主管）阐述，“基于美国、加拿大和欧美健康监管当局的反馈意见，我们相信BENEFIT-ALS项目可能对tirasemtiv成为潜在的获批ALS治疗药物有支持作用。”
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