A multi-center pivotal field study to confirm the effectiveness and safety of verdinexor for the treatment of lymphoma in dogs.

一项多中心关键现场研究，旨在确认 verdinexor 治疗犬淋巴瘤的有效性和安全性。

• 批准号: 10159259
• 负责人: David Bruyette
• 金额: $ 25万
• 依托单位: ANIVIVE LIFESCIENCES, INC
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-06-01 至 2023-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10159259
• 关键词: multi; center; pivotal; field; study

Project Summary/Abstract: The proposed study will contribute to completing the requirements for our New Animal Drug Application (NADA) effectiveness technical section for the designated intended use. Study Design This will be a prospective, randomized, double-masked, placebo-controlled, multi-site, pivotal field study designed to evaluate verdinexor for the treatment of lymphoma in dogs. Treatment Groups and Number of Animals On study Day 0, study-eligible dogs will be randomized to the IVP or CP treatment group in a 4:1 ratio, in blocks of 5. A single study protocol will be followed at multiple study locations. A sufficient number of dogs will be enrolled in the study across all sites to complete the study with at least 100 evaluable cases treated with verdinexor and 25 evaluable cases treated with placebo tablets. An evaluable sample size of 100 IVP and 25 CP dogs, assuming a median time to progression of 28 days and 7 days respectively, will provide more than 90% power (alpha = 0.05, 2-sided). To be eligible for the statistical analysis, each study site must complete a minimum of 5 evaluable cases, at least 1 per treatment group. No more than 25% of evaluable cases should be enrolled at any one site. A simulation of 10,000 clinical trials, using the statistical model specified for the analysis, was generated to determine if the proposed sample size would have adequate power. Statistical Analysis: Effectiveness Endpoints: Primary Effectiveness Variable The primary effectiveness variable will be time to progression (TTP). TTP is defined as the time from Day 0 to time of progressive disease. A case will be considered to be in progressive disease state if either the definition of progressive disease for target lesions in section 16.8.1.3, OR, if the definition of progressive disease for non-target lesions in section 16.8.2.2, is met. Effectiveness will be established if the median TTP of lymphoma in dogs is statistically significantly longer in the treated group compared to the placebo group. Correlation of the statistical treatment success to the clinical benefit of the drug will be addressed in the Final Study Report (FSR). Safety Assessment Safety will be evaluated based on physical exam, serum chemistry, hematology and urinalysis parameters and the occurrence of adverse events.

项目摘要/摘要： 拟议的研究将有助于完成我们的新兽药的要求 指定预期用途的应用（NADA）有效性技术部分。 研究设计 这将是一项前瞻性、随机、双盲、安慰剂对照、多部位、关键的研究 旨在评估 verdinexor 治疗犬淋巴瘤的实地研究。 治疗组和动物数量 在研究第 0 天，符合研究条件的狗将按 4:1 随机分配到 IVP 或 CP 治疗组 比率，以 5 为单位。将在多个研究地点遵循单一研究方案。一个 所有地点将有足够数量的狗参加研究以完成研究 至少有 100 例使用 verdinexor 治疗的可评估病例和 25 例使用 Verdinexor 治疗的可评估病例 安慰剂片剂。假设中位时间，可评估样本量为 100 只 IVP 狗和 25 只 CP 狗 分别进行 28 天和 7 天，将提供超过 90% 的功率（alpha = 0.05，2 面）。为了有资格进行统计分析，每个研究中心必须完成一份 至少 5 个可评估病例，每个治疗组至少 1 个。不超过可评估的25% 病例应在任一地点登记。模拟 10,000 次临床试验，使用 生成为分析指定的统计模型，以确定建议的样本是否 尺寸就有足够的功率。 统计分析：有效性终点：主要有效性变量 主要有效性变量是进展时间（TTP）。 TTP 定义为时间 从第 0 天到疾病进展时。案件将被视为正在进行中 疾病状态如果符合第 16.8.1.3 节中目标病变的进行性疾病的定义， 或者，如果满足第 16.8.2.2 节中非目标病变的进展性疾病的定义。 如果对犬淋巴瘤的中位 TTP 进行统计，则可以确定有效性 与安慰剂组相比，治疗组的时间明显更长。的相关性 统计治疗成功与否对药物临床效益的影响将在决赛中讨论 研究报告（FSR）。 安全评估 将根据体检、血清化学、血液学和尿液分析评估安全性 参数和不良事件的发生。