Competitive Renewal for Multicenter Uveitis Steroid Treatment (MUST) Trial

多中心葡萄膜炎类固醇治疗 (MUST) 试验的竞争性续展

• 批准号: 8843859
• 负责人: Janet T. Holbrook
• 金额: $ 153.59万
• 依托单位: JOHNS HOPKINS UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2004
• 资助国家: 美国
• 起止时间: 2004-07-15 至 2016-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8843859
• 关键词: Adrenal Cortex Hormones; Adverse effects; Agreement; Autoimmune Process; Blindness; Cataract; Chairperson; Choroidal Neovascularization; Clinical; Clinical Management; Clinical Research; Clinical Treatment; Clinical Trials; Clinical Trials Network; Collaborations; Consultations; Data; Data Collection; Data Quality; Disease; Effectiveness; Epidemiologic Studies; Epiretinal Membrane; Evaluation; Fluocinolone Acetonide; Glaucoma; Goals; Group Structure; Human Resources; Humira; Immunosuppression; Immunosuppressive Agents; Implant; Inflammation; Laboratories; Local Therapy; Lucentis; Methods; Monitor; Monoclonal Antibodies; Online Systems; Oral; Outcomes Research; Participant; Patient Agents; Patients; Performance; Pharmaceutical Preparations; Pharmacotherapy; Pilot Projects; Principal Investigator; Procedures; Process; Protocols documentation; Publications; Quality Control; Randomized Controlled Clinical Trials; Reading; Recruitment Activity; Refractory; Relative (related person); Reporting; Research; Research Personnel; Retinal Neovascularization; Rheumatoid Arthritis; Safety; San Francisco; Series; Slide; Steroids; Strategic Planning; System; Systemic Therapy; Time; Training; Triamcinolone Acetonide; Tumor Necrosis Factor-alpha; United States Food and Drug Administration; Universities; Update; Uveitis; Vascular Endothelial Growth Factors; Vision; Wisconsin; Work; adalimumab; cohort; comparative trial; compare effectiveness; data management; design; effective therapy; evidence base; follow-up; high risk; human monoclonal antibodies; macular edema; medical schools; primary outcome; quality assurance; ranibizumab; research study; standard care; treatment trial; trial comparing; web site

DESCRIPTION (provided by applicant): The goal of this proposal is to develop a clinical trials network capable of engaging in several clinical trials of the treatments of uveitis and its complications. The MUST Research Group (RG) structure already is in place, and processes have been developed for review, approval, and prioritization of clinical trials. Four clinical research studies are proposed as the initial activities of the proposed network, which represent the specific aims of the proposal. These are: 1) To continue the current MUST Trial follow-up past the original two-year determination of the primary outcome for an additional 6 years. This long-term follow-up will enable the MUST RG to evaluate the long-term consequences of the two treatment paradigms: regional corticosteroid treatment v. systemic therapy with corticosteroids and immunosuppression; 2) To compare the relative effectiveness of adalimumab (Humira(R). Abbott Laboratories. Abbott Park. IL). a fully human monoclonal antibody to TNF-a v. conventional immunosuppression for patients with severe uveitis requiring immunosuppressive drug therapy. Adalimumab already is approved by the US Food and Drug Administration (FDA) for the treatment of several autoimmune or autoinflammatory diseases, such as rheumatoid arthritis, and preliminary data suggest that it is effective as a corticosteroid-sparing agent in patients with uveitis; 3) To compare periocular v. intravitreal corticosteroids for the treatment of macular edema in patients with uveitis. Both periocular and intravitreal corticosteroids appear to be effective for the treatment of macular edema in patients with uveitis, but no comparative trials have been performed. Many clinicians believe that intravitreal corticosteroids are more effective than periocular corticosteroids for this indication, but that they also have higher rates of side effects; and 4) To compare intravitreal ranibizumab (Lucentis(R). Genentech Inc., San Francisco. CA). a monoclonal antibody to vascular endothelial growth factor, to intravitreal corticosteroids for the treatment of refractory macular edema in patients with uveitis. A pilot study suggested that ranibizumab was effective in the treatment of uveitic macular edema and that potentially it may have less ocular side effects than corticosteroids. However, the relative efficacy of the two treatments is unknown. RELEVANCE: Non-infectious uveitis is associated with high rates of visual loss and usually requires long-term therapy, often with oral corticosteroids, supplemented, when indicated, by immunosuppressive (corticosteroid- sparing) drugs. Visual loss often is caused by structural complications, e.g.. macular edema. We propose to carry out a series of clinical studies to provide an evidence base for the management of these conditions.

描述(由申请人提供)：这项建议的目标是发展一个临床试验网络，能够从事葡萄膜炎及其并发症的治疗的几个临床试验。必须研究小组(RG)的结构已经到位，并且已经制定了临床试验的审查、批准和优先顺序的程序。四项临床研究研究被提议作为拟议网络的初始活动，它们代表了提议的具体目标。这些是：1)要继续目前的试验，必须在最初的两年初步结果确定之后再进行6年的后续试验。这一长期随访将使MASH RG能够评估两种治疗模式的长期结果：局部皮质类固醇治疗与全身皮质类固醇治疗和免疫抑制；2)比较阿达利单抗(Humira(R))的相对有效性。雅培实验室。雅培公园。Il)。针对需要免疫抑制药物治疗的严重葡萄膜炎患者的常规免疫抑制治疗。Adalimumab已经被美国食品和药物管理局(FDA)批准用于治疗几种自身免疫性或自身炎症性疾病，如类风湿性关节炎，初步数据表明，它作为葡萄膜炎患者的皮质类固醇节约剂是有效的；3)比较眼周和玻璃体内皮质类固醇治疗葡萄膜炎患者的黄斑水肿。眼周和玻璃体内的皮质类固醇似乎都能有效地治疗葡萄膜炎患者的黄斑水肿，但还没有进行对比试验。许多临床医生认为，对于这一适应症，玻璃体内的皮质类固醇比眼周皮质类固醇更有效，但它们的副作用也更高；4)比较玻璃体内的ranibizumab(Lucentis(R))。基因技术公司，旧金山。CA)。抗血管内皮生长因子、玻璃体内皮质类固醇的单抗用于治疗葡萄膜炎患者的顽固性黄斑水肿。一项初步研究表明，雷尼比珠单抗在治疗葡萄膜炎黄斑水肿方面有效，可能比皮质类固醇有更少的眼部副作用。然而，这两种疗法的相对疗效尚不清楚。 相关性：非感染性葡萄膜炎与高视力损失率相关，通常需要长期治疗，通常是口服皮质类固醇，必要时辅以免疫抑制(保留皮质类固醇)药物。视力丧失通常是由结构并发症引起的，例如。黄斑水肿。我们建议开展一系列临床研究，为这些疾病的治疗提供证据基础。