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: 10086617
• 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 2022-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10086617
• 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.

项目概要/摘要： 拟议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、随机化、双盲、安慰剂对照、多中心、关键性 旨在评价verdinexor治疗犬淋巴瘤的田间研究。 给药组和动物数量 在研究第0天，符合研究资格的犬将以4：1的比例随机分配至IVP或CP治疗组 比例，以5为单位。多个研究中心将遵循单一研究方案。一 所有研究中心将入组足够数量的犬以完成研究 至少100例可评价病例接受verdinexor治疗，25例可评价病例接受 安慰剂片剂。可评价样本量为100只IVP犬和25只CP犬，假设中位时间 分别为28天和7天的进展，将提供超过90%的把握度（α = 0.05，双侧）。为了符合统计分析的资格，每个研究中心必须完成 至少5例可评价病例，每个治疗组至少1例。不超过可评价的25% 病例应在任何一个研究中心入组。模拟10，000个临床试验，使用 生成用于分析的指定统计模型，以确定拟定样品是否 大小将具有足够的功率。 统计分析：有效性终点：主要有效性变量 主要有效性变量为至疾病进展时间（TTP）。TTP定义为时间 从第0天至疾病进展时间。一个案件将被认为是在进行中 疾病状态，如果是第16.8.1.3节中靶病变的疾病进展定义， 或者，如果符合第16.8.2.2节中非靶病灶疾病进展的定义。 如果犬淋巴瘤的中位TTP在统计学上 与安慰剂组相比，治疗组的时间显著更长。相关性 统计治疗成功对药物临床获益的影响将在最终报告中讨论。 研究报告（FSR）。 安全评估 将根据体格检查、血清生化、血液学和尿分析评价安全性 参数和不良事件的发生。