A Scientific Meeting of ALS Clinical Trials

ALS 临床试验科学会议

• 批准号: 6668799
• 负责人: HIROSHI MITSUMOTO
• 金额: $ 3.5万
• 依托单位: COLUMBIA UNIVERSITY HEALTH SCIENCES
• 依托单位国家: 美国
• 财政年份: 2003
• 资助国家: 美国
• 起止时间: 2003-06-09 至 2004-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6668799
• 关键词: amyotrophic lateral sclerosis; clinical trials; meeting /conference /symposium; neural degeneration

DESCRIPTION (provided by the applicant): Amyotrophic Lateral Sclerosis (ALS) is one of the most devastating neurodegenerative disorders, leading to death 3-5 years after symptom onset. Approved since 1996, Riluzole is the only drug available for ALS, but its benefit is limited. Clinical trials of all other potential ALS drugs in the past decade have failed. Current rapid progress in translational research is likely to make an increasing number of novel therapies available for clinical trials in ALS. Thus, ALS clinical trialists must be prepared to conduct much more effective and efficient clinical trials in the near future. We propose to hold a scientific meeting on ALS clinical trials at Tarrytown House, Westchester, New York, on June 13-15, 2003. The goals of this meeting are: 1) expanding the knowledge base in regard to clinical trials for both experienced and future clinical trialists in ALS; 2) generating new clinical trial designs through discussion of controversies, new ideas, and new directions; and 3) evaluating the current collaborative study groups and examining new ideas for a national collaborative study group. On Day 1, we will focus on the educational component, discussing translational research that is producing novel therapies, current issues in clinical trials, in general, and lessons from trials in diseases other than ALS. Day 2 will focus on 6 topics crucial for developing effective and efficient future ALS trials: 1) problems in defining the natural history of ALS; 2) selecting primary outcome measures; 3) protecting patients and preserving trial quality; 4) study designs and aims; 5) emerging surrogate markers; and 6) drug combination trials. Day 3 will focus on funding issues, the issues of the clinical trial community, and the pros and cons of national and regional study groups as effective collaborative structures. The participants will include ALS clinical trialists, basic research scientists, young clinical investigators/fellows, funding agencies, regulatory agencies, drug companies, and anyone who has a significant interest in ALS clinical trials. The proposed meeting will be the first NIH-supported scientific meeting in ALS clinical trials, providing an unprecedented opportunity to discuss issues that must be resolved so that clinical trialists can conduct more effective and sophisticated trials that make the best use of advances in translational research, and create the greatest opportunities to identify effective new drugs in ALS.

描述（由申请人提供）：肌萎缩性侧索硬化症（ALS）是最具破坏性的神经退行性疾病之一，在症状出现后3-5年导致死亡。自1996年批准以来，利鲁唑是唯一可用于ALS的药物，但其益处有限。在过去的十年中，所有其他潜在的ALS药物的临床试验都失败了。目前转化研究的快速进展可能会使越来越多的新疗法可用于ALS的临床试验。因此，ALS临床试验人员必须准备在不久的将来进行更有效和高效的临床试验。我们建议于2003年6月13日至15日在纽约州威彻斯特的Tarrytown House举行一次关于ALS临床试验的科学会议。本次会议的目标是：1)为ALS临床试验的经验和未来的临床试验人员扩大有关临床试验的知识库；2)通过讨论争议、新观点和新方向，产生新的临床试验设计；3)评估当前的合作学习小组，并研究建立全国性合作学习小组的新思路。在第一天，我们将重点关注教育部分，讨论产生新疗法的转化研究，临床试验中的当前问题，以及ALS以外疾病试验的经验教训。第2天将集中讨论6个对开发有效和高效的未来ALS试验至关重要的主题：1)定义ALS自然历史的问题；2)选择主要结局指标；3)保护患者，保证试验质量；4)研究设计和目的；5)新兴的替代标记物；6)药物联合试验。第3天将重点讨论资金问题、临床试验社区的问题，以及国家和区域研究小组作为有效合作结构的利弊。参与者将包括ALS临床试验人员、基础研究科学家、年轻临床研究者/研究员、资助机构、监管机构、制药公司以及任何对ALS临床试验有重大兴趣的人。这次会议将是美国国立卫生研究院支持的第一次ALS临床试验科学会议，提供了一个前所未有的机会来讨论必须解决的问题，以便临床试验学家能够进行更有效和更复杂的试验，充分利用转化研究的进展，并创造最大的机会来识别有效的ALS新药。