AMD Treatment Network Chairman Clinical Study Planning

在美国和大多数发达国家，老年性黄斑变性是导致失明的主要原因。对AMD患者的新治疗方法进行评估的必要性很大，而且可能会持续多年。尽管最近报道了维替泊芬的光动力疗法(PDT)的好处，但对于新生血管性AMD患者来说，仍然非常需要额外的治疗方法。许多AMD患者没有资格接受PDT，治疗益处的有限和PDT的高昂成本留下了很大的改进空间。越来越多的人达成共识，认为联合治疗将是必要的，以提供真正的保护，防止严重的视力损失。目前还没有经过验证的治疗地理萎缩的方法。如果年龄相关眼病研究(AREDS)和AMD预防试验(CAPT)的结果显示对膳食补充剂和/或预防性激光治疗有好处，保护作用可能是中等的，留有改进的空间。 这项建议的目标是： 1)建立基础设施，加快开发和进行治疗和预防老年性黄斑变性的临床试验。拟议的网络将协助合作研究人员与NEI工作人员合作，为多中心临床试验制定和实施具体、详细的方案。 2)为一项或多项临床试验制定详细的方案，这将是第一次使用该基础设施。 该网络的主席将负责仔细审查临床试验的所有潜在领域，并评价其重要性和及时性。在规划过程中，他将与实地的其他领导人，包括NEI的内部工作人员进行磋商。

Age-related macular degeneration is the leading cause of blindness in the United States and most developed countries. The need for evaluation of new treatment approaches for patients with AMD is great and likely to continue for many years. Despite the recently documented benefits of photodynamic therapy (PDT) with verteporfin, there is still great need for additional treatment approaches for patients with neovascular AMD. Many patients with AMD are not eligible for PDT and the limited magnitude of treatment benefit and the high cost of PDT leave much room for improvement. There is a growing consensus that combination therapies will be necessary to provide true protection from severe visual acuity loss. There are no proven treatments for geographic atrophy. If the results of the Age-Related Eye Disease Study (AREDS) and the Complications of AMD Prevention Trial (CAPT) show benefit to dietary supplements and/or prophylactic laser treatment, the protective effects are likely to be moderate, leaving room for improvement. The objectives of this proposal are: 1) To develop an infrastructure to accelerate the development and conduct of clinical trials of the treatment and prevention of age-related macular degeneration. The proposed network would assist collaborating investigators, working in partnership with NEI staff, in developing and implementing specific, detailed protocols for multicenter clinical trials. 2) To develop a detailed protocol for one or more clinical trials that would be the first to use the infrastructure. The Chair of the Network will be responsible for carefully reviewing the full range of potential areas for clinical trials and evaluating their importance and timeliness. He will confer with other leaders in the field, including the intramural staff of the NEI during the planning process.