NAC Attack, a phase-3, multicenter, randomized, placebo-controlled trial in patients with retinitis pigmentosa

摘要：视网膜色素变性是一种遗传性视网膜变性，由一种或多种 基因突变的数量。它是导致20-60岁人群失明和严重视力丧失的主要原因。 好几年了。RP的首发症状是由突变导致的视杆细胞死亡所致的夜视丧失。 光感受器。随后视锥感光细胞的丧失会导致视野逐渐缩小和 最终导致失明。目前，还没有预防RP患者视力丧失的治疗方法。NAC攻击是第3阶段 多中心、随机、双盲、平行、安慰剂对照临床试验评价疗效 口服N-乙酰半胱氨酸(NAC)对RP患者的安全性。参与者将被随机分配到 口服NAC 1800毫克/次或安慰剂，比例为2：1，并将随访45个月。 协调中心(CC)与执行委员会密切合作，以确保成功 南汽攻击的证据。CC有助于研究领导力，并提供试验设计、促进 征聘和留用参与者、协调所有审判实体、实施和维护 高质量的数据管理系统、统计分析和质量保证。消委会将： 招募和保留临床站点，拥有经验丰富的研究人员和足够的招聘能力； 确保临床现场人员完成认证要求并遵守法规； 通过设计和实现数据采集表单，创建和维护学习数据库，安全 使用RedCap的基于Web的数据捕获、数据编辑和数据管理； 使用JHU OneDrive创建和维护基于研究的云门户，以接收和管理映像和 临床现场的测试文件； 就实时问题的解决和成功实施的总体策略向临床站点提供建议 研究方案的内容； 为临床站点的研究管理提供帮助，如预约时间表、即将到来的提醒 探访、遗漏表格、提交学习材料不完整； 定期向临床站点和所有研究委员会提供研究进展和绩效报告； 设计并实施完整的质量保证活动计划，包括人员认证和 临床站点、现场访问和性能监控； 提供研究数据的中期和最终统计分析； 筹备各种委员会会议和研究会议； 参与并领导科学报告和报告的准备工作。 NAC攻击有可能确定一种使所有RP患者受益的药物疗法，无论 对其致病突变的鉴定，从而影响RP的临床治疗。

ABSTRACT: Retinitis Pigmentosa (RP) is an inherited retinal degeneration caused by one or more of a large number of genetic mutations. It is a major cause of blindness and severe vision loss in people aged 20-60 years. The first symptom in RP is loss of night vision as a result of mutation-caused death of rod photoreceptors. Subsequent loss of cone photoreceptors leads to gradual constriction of visual field and eventually blindness. Currently, there is no treatment for preventing vision loss in RP. NAC Attack is a phase-3 multicenter, randomized, double masked, parallel and placebo-controlled clinical trial to evaluate the efficacy and safety of oral N-acetylcysteine (NAC) in patients with RP. Participants will be randomized to receive either oral NAC of 1800mg/bid or placebo in a ratio of 2:1 and will be followed for 45-months. The Coordinating Center (CC) collaborates closely with the Executive Committee to ensure the success of NAC Attack. The CC contributes to study leadership and provides expertise on trial design, facilitation of recruitment and retention of participants, coordination across all trial entities, implementation and maintenance of a high-quality data management system, statistical analysis, and quality assurance. The CC will:  Enlist and retain Clinical Sites with experienced investigators and sufficient recruitment capacity;  Ensure that Clinical Site personnel complete certification requirements and are in regulatory compliance;  Create and maintain the study database through design and implementation of data collection forms, secure web-based data capture using REDCap, data editing, and data management;  Create and maintain the study cloud-based portal using JHU OneDrive to receive and manage imaging and test files from the clinical sites;  Advise Clinical Sites on resolution of real-time problems and overall strategies for successful implementation of the study protocol;  Provide aids to clinical sites for study management such as appointment schedule, reminders of upcoming visits, missing forms, and incomplete submission of study materials;  Provide regular reports on study progress & performance to Clinical Sites and all study committees;  Design and implement a full program of quality assurance activities including certification of personnel and clinical sites, site visits, and performance monitoring;  Provide interim and final statistical analyses of study data;  Prepare for various committee and study meetings;  Participate and lead in the preparation of scientific presentations and reports. NAC Attack has the potential to identify a pharmacological therapy benefitting all patients with RP, irrespective of the identification of their causative mutation, and thus to impact on the clinical management of RP.