Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study - COVID supplement

随机、双盲、安慰剂对照、第 3 期研究 - COVID 补充

• 批准号: 10610578
• 负责人: Susan Abushakra
• 金额: $ 350万
• 依托单位: ALZHEON, INC.
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-08-15 至 2025-05-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10610578
• 关键词: Address; Administrative Supplement; Alzheimer' s Disease; Amyloid; Biological Markers; Brain; COVID-19 impact; COVID-19 pandemic; Canada; Clinical Research; Contracts; Country; Czech Republic; Double-Blind Method; Evaluation; France; Genotype; Germany; Goals; Iceland; Institution; Investments; Laboratories; Liquid substance; Magnetic Resonance Imaging; Nerve Degeneration; Netherlands; Oral; Patients; Phase; Placebo Control; Placebos; Randomized; Safety; Site; Site Visit; Spain; Sum; United Kingdom; United States National Institutes of Health; arm; coronavirus disease; cost; efficacy evaluation; efficacy study; outreach; pandemic disease; phase 3 study; phase III trial; placebo controlled study; recruit; safety study; screening; small molecule

Project Summary We are developing ALZ-801 (also known as valiltramiprosate), an oral brain-penetrant small molecule that inhibits the formation of toxic amyloid oligomers. With support from NIA through R01-AG065253, we are conducting a Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy, Safety and Biomarker Effects of ALZ-801 in Subjects with Early Alzheimer’s Disease and APOE4/4 Genotype (NCT04770220). This study (ALZ-801-AD301, APOLLOE4) evaluates the efficacy, safety, fluid biomarkers, and MRI effects of ALZ-801 265 mg bid or placebo in 300 patients over 78 weeks (150/arm). The study is being conducted in the US, Canada, Iceland, United Kingdom, Czech Republic, Netherlands, Germany, France, and Spain. A major clinical research organization (CRO), ICON plc, is contracted to manage the study. Study start-up commenced in July 2020, in the middle of the first year of the COVID-19 pandemic and the first subject was randomized in June 2021. The original goal for the study was to complete randomization within 18 months (i.e., by October 2022) with a goal of completing the study by May 2024. Due to the complications of the pandemic, the study has faced many complications that have increased both the cost and duration of the study: • many sites initially chosen for the study were unable to participate or were severely delayed in participation due to COVID as many sites were unable to conduct on-site visits or were unable to see potential new subjects for evaluation and recruitment – as a result an additional 14 sites are being brought to site initiation and a new country (Czech Republic) was added to the study • especially at some of the larger institutions, administrative support was focused on COVID studies and contracting was for other studies was severely delayed • enhancement or initiation of alternate strategies for recruitment were employed to address the very limitation application of traditional, local outreach strategies • supply chain issues have lately had a significant impact, especially on the provision of laboratory supplies, particularly kits to support screening activities causing some sites to pause screening activities The sum of these challenges has resulted in a delay of approximately two months in recruitment with subsequent increases in project management. This delay would likely have been significantly longer without the increase in investment already applied to the study by the sponsor.

项目概要 我们正在开发 ALZ-801（也称为valiltramiprosate），这是一种口服脑渗透性小分子， 抑制有毒淀粉样蛋白寡聚体的形成 在 NIA 通过 R01-AG065253 的支持下，我们 进行有效性、安全性的 3 期、多中心、随机、双盲、安慰剂对照研究 ALZ-801 对早期阿尔茨海默病和 APOE4/4 基因型受试者的生物标志物影响 (NCT04770220)。本研究（ALZ-801-AD301、APOLLOE4）评估了疗效、安全性、液体生物标志物和 ALZ-801 265 mg bid 或安慰剂在 78 周内对 300 名患者（150 名/组）进行的 MRI 效果该研究正在进行中。 在美国、加拿大、冰岛、英国、捷克共和国、荷兰、德国、法国和 西班牙主要临床研究组织 (CRO) ICON plc 签约管理该研究。 研究启动于 2020 年 7 月，即 COVID-19 大流行的第一年中期和第一个 受试者于 2021 年 6 月进行随机分组。该研究的最初目标是在 18 年内完成随机分组 几个月（即 2022 年 10 月之前），目标是在 2024 年 5 月之前完成研究。由于研究的复杂性 大流行期间，该研究面临许多并发症，增加了研究的成本和持续时间： • 许多最初选择的研究地点无法参与或严重延迟参与 由于新冠疫情，许多网站无法进行现场访问或无法看到潜在的新产品 评估和招募对象——因此，另外 14 个站点被纳入站点启动阶段 研究中添加了一个新国家（捷克共和国） • 特别是在一些较大的机构，行政支持主要集中在新冠病毒研究和 其他研究的合同被严重延误 • 采用增强或启动替代招募策略来解决这一限制 应用传统的本地推广策略 • 供应链问题最近产生了重大影响，特别是在实验室用品的供应方面， 特别是支持筛查活动的套件，导致一些站点暂停筛查活动 这些挑战的总和导致招聘延迟了大约两个月 如果项目管理的增加，这种延迟可能会显着延长。 赞助商已将投资应用于该研究。