Chair Grant for the Comparison for AMD Treatment Trials

描述(申请人提供)：2006年9月，国家眼科研究所资助了AMD治疗试验比较(CATT)。这项有1200名患者参加的4组研究旨在确定Lucentis和Avestin的相对疗效，并评估它们的最佳剂量频率。患者登记原计划在2007年初开始，然而，监管障碍，包括CMS为这些代理获得报销，将试验期限延长到目前的资金之外。此外，很明显，超过两年的长期视觉结果包括安全性和有效性是完全未知的。鉴于Lucentis和阿瓦斯丁现在是全世界几乎所有AMD治疗的基础，而且这些患者需要在之前的研究阶段之后的许多年里继续服用，显然必须了解这些药物的长期效果。在本申请中，请求支持两项计划：具体目标1：完成CATT：Lucentis-Avestin随机临床试验。本期供资将于2010年8月结束。最后一名登记的患者将于2011年12月完成他们的两年疗程。需要资金以确保所有患者在完成CATT研究的第一阶段时得到支持。具体目标#2：进行一项观察性随访研究，以调查长期使用Lucentis或阿瓦斯丁治疗的视力进程以及眼部和全身的安全性。在随机临床试验中完成两年治疗的患者将在观察性研究中再跟踪三年。相关性(参见说明书)：Lucentis和阿瓦斯丁的开发代表了对AMD以前疗法的重大治疗改进，并深刻改变了人们对可以预防或逆转视力丧失程度的预期。没有一项研究对这两种药物的安全性和有效性进行正面比较，也没有一项研究观察了这两种药物按月或按需给药方案的比较结果。

DESCRIPTION (provided by applicant): In September, 2006, the National Eye Institute funded the Comparison of AMD Treatments Trials (CATT). This 4-arm, 1200 patient study was designed to determine the relative efficacy of Lucentis and Avastin and to evaluate their optimal dosing frequencies. Patient enrollment was planned to have begun in early 2007, however, regulatory hurdles including obtaining reimbursement for these agents by CMS extended the period of the trial beyond its current funding. In addition, it has become clear that the long term visual outcomes including safety and efficacy beyond two years are completely unknown. Given that Lucentis and Avastin are now the basis for virtually all AMD treatment worldwide and that these patients require continued dosing for many years beyond the previous periods of study, it is clear that the long term effects of these drugs must be understood. In this application, support for two initiatives is requested: Specific Aim #1: To complete the CATT: Lucentis-Avastin randomized clinical trial. The current period of funding ends August 2010. The last patient enrolled will complete their two year period of treatment in December 2011. Funding is requested to insure all patients are supported through completion of the first phase of the CATT study. Specific Aim #2: To conduct an observational Follow-up Study to investigate the course of visual acuity and the ocular and systemic safety of long-term treatment with Lucentis or Avastin. Patients who complete two years of treatment in the randomized clinical trial will be followed in an observational study for an additional three years. RELEVANCE (See instructions): The development of Lucentis and Avastin represents a major therapeutic improvement over previous therapies for AMD and has profoundly altered expectations regarding the extent of visual loss that can be prevented or reversed. No single study has compared the safety and efficacy of these two agents head to head or looked at comparative outcomes of these agents using monthly or "as needed" dosing regimens.