Centre for Rheumatic Diseases, Department of Inflammation Biology, School of Immunology and Microbial Sciences, Faculty of Life Sciences and Medicine, King's College London, Weston Education Centre, 10 Cutcombe Road, London, SE5 9RJ, UK.
Department of Rheumatology, National Institute for Health Research-Wellcome Clinical Research Facility, Hammersmith Hospital, Imperial College, London, W12 0HS, UK.
Trials. 2019 Jul 15;20(1):429. doi: 10.1186/s13063-019-3403-7.
We present a study protocol for a multi-centre, randomised, double-blind, parallel-group, placebo-controlled trial that seeks to test the feasibility, acceptability and effectiveness of a 52-week period of treatment with the first-in-class co-stimulatory blocker abatacept for preventing or delaying the onset of inflammatory arthritis.
The study aimed to recruit 206 male or female subjects from the secondary care hospital setting across the UK and the Netherlands. Participants who were at least 18 years old, who reported inflammatory sounding joint pain (clinically suspicious arthralgia) and who were found to be positive for serum autoantibodies associated with rheumatoid arthritis (RA) were eligible for enrolment. All study subjects were randomly assigned to receive weekly injections of investigational medicinal product, either abatacept or placebo treatment over the course of a 52-week period. Participants were followed up for a further 52 weeks. The primary endpoint was defined as the time to development of at least three swollen joints or to the fulfilment of the 2010 American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) classification criteria for RA using swollen but not tender joints, whichever endpoint was met first. In either case, swollen joints were confirmed by ultrasonography. Participants, care givers, and those assessing the outcomes were all blinded to group assignment. Clinical assessors and ultrasonographers were also blinded to each other's assessments for the duration of the study.
There is limited experience of the design and implementation of trials for the prevention of inflammatory joint diseases. We discuss the rationale behind choice and duration of treatment and the challenges associated with defining the "at risk" state and offer pragmatic solutions in the protocol to enrolling subjects at risk of RA.
Current Controlled Trials, ID: ISRCTN46017566 . Registered on 4 July 2014.
我们提出了一项多中心、随机、双盲、平行组、安慰剂对照试验的研究方案，旨在测试新型共刺激阻断剂阿巴西普治疗 52 周，预防或延缓炎症性关节炎发病的可行性、可接受性和有效性。
该研究旨在从英国和荷兰的二级保健医院招募 206 名男性或女性受试者。年龄至少 18 岁、报告有炎症性关节痛（临床可疑关节炎）且血清自身抗体阳性与类风湿关节炎（RA）相关的患者有资格入组。所有研究对象均随机分配接受每周一次的研究药物治疗，即阿巴西普或安慰剂治疗，持续 52 周。参与者随访 52 周。主要终点定义为出现至少 3 个肿胀关节的时间，或满足 2010 年美国风湿病学会/欧洲抗风湿病联盟（ACR/EULAR）RA 分类标准的时间，以先满足的为准。在任何一种情况下，肿胀关节均通过超声确认。参与者、护理人员和评估结局的人员均对分组情况设盲。临床评估人员和超声医师在整个研究期间也对彼此的评估设盲。
预防炎症性关节疾病的试验设计和实施经验有限。我们讨论了选择和治疗持续时间的理由，以及定义“高危”状态所面临的挑战，并在方案中提供了实用的解决方案，以招募有患 RA 风险的受试者。
当前对照试验，ID：ISRCTN46017566。于 2014 年 7 月 4 日注册。
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