Chair Grant for the Comparison for AMD Treatment Trials

描述（由申请人提供）：2006年9月，国家眼科研究所资助了AMD治疗试验比较（CATT）。这项4组1200例患者的研究旨在确定Lucentis和Avastin的相对疗效，并评估它们的最佳给药频率。患者登记计划在2007年初开始，然而，包括获得CMS对这些药物的报销在内的监管障碍延长了试验的期限，超出了目前的资助。此外，很明显，长期的视力结果，包括两年以上的安全性和有效性，是完全未知的。鉴于Lucentis和Avastin现在是全世界几乎所有AMD治疗的基础，并且这些患者需要在之前的研究期之外持续服用多年，很明显，必须了解这些药物的长期影响。在此申请中，要求支持两个倡议：具体目标#1：完成CATT: Lucentis-Avastin随机临床试验。目前的资助期将于2010年8月结束。最后一名患者将于2011年12月完成为期两年的治疗。申请资金以确保所有患者完成第一阶段的CATT研究。具体目标2：开展一项观察性随访研究，调查Lucentis或Avastin长期治疗的视力过程和眼部和全身安全性。在随机临床试验中完成两年治疗的患者将在另一项观察性研究中随访三年。相关性（见说明书）：Lucentis和Avastin的开发代表了AMD治疗方法的重大改进，并深刻地改变了人们对可预防或逆转视力丧失程度的期望。没有单独的研究比较了这两种药物的安全性和有效性，也没有观察到这些药物使用每月或“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.