A Scientific Meeting of ALS Clinical Trials

描述(由申请人提供)：肌萎缩侧索硬化症(ALS)是最具破坏性的神经退行性疾病之一，导致症状出现3-5年后死亡。自1996年获得批准以来，利鲁唑是唯一可用于治疗ALS的药物，但其疗效有限。在过去的十年里，所有其他潜在的ALS药物的临床试验都失败了。目前翻译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临床试验人员必须做好准备，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13-15日在纽约韦斯特切斯特的Tarrytown House举行一次关于ALS临床试验的科学会议。这次会议的目标是：1)为有经验的和未来的ALS临床试验人员扩大临床试验的知识库；2)通过讨论争议、新想法和新方向来产生新的临床试验设计；以及3)评估当前的合作研究小组，并审查国家合作研究小组的新想法。在第一天，我们将集中讨论教育部分，讨论正在产生新疗法的转化性研究，临床试验中的当前问题，以及ALS以外的疾病试验的经验教训。第二天将集中讨论对开发有效和高效的未来肌萎缩侧索硬化症试验至关重要的6个主题：1)定义肌萎缩侧索硬化症的自然历史的问题；2)选择主要结果指标；3)保护患者和保持试验质量；4)研究设计和目标；5)新出现的替代标记物；以及6)药物组合试验。第三天将集中讨论资金问题、临床试验社区的问题，以及作为有效合作结构的国家和地区研究小组的利弊。参与者将包括肌萎缩侧索硬化症临床试验者、基础研究科学家、年轻的临床研究人员/研究员、资助机构、监管机构、制药公司，以及任何对肌萎缩侧索硬化症临床试验感兴趣的人。拟议中的会议将是NIH支持的ALS临床试验中的第一次科学会议，提供了一个前所未有的机会来讨论必须解决的问题，以便临床试验人员能够进行更有效和更复杂的试验，最大限度地利用翻译研究的进展，并创造最大的机会来确定ALS有效的新药。

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.