INCLUSION BODY MYOSITIS: PILOT STUDY OF B-INTERFERONLA

包涵体肌炎：B-干扰素的试点研究

• 批准号: 6187401
• 负责人: RABI TAWIL
• 金额: $ 44.43万
• 依托单位: UNIVERSITY OF ROCHESTER
• 依托单位国家: 美国
• 财政年份: 1999
• 资助国家: 美国
• 起止时间: 1999-09-23 至 2002-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6187401
• 关键词: clinical research; clinical trials; human subject; human therapy evaluation; inclusion body; interferon beta; muscle disorder chemotherapy; muscle strength; myositis

Inclusion body myositis (IBM), is the most common acquired disease of muscle in adults over the age of 50. IBM has a typical clinical presentation but requires muscle biopsy for definitive diagnosis. Although, there is compelling evidence for the importance of immunologic abnormalities in it pathogenesis, the cause of the disease is not known and it is generally considered to be resistant to treatment with conventional immunosuppressive agents. Beta interferon-1a (betaINF1a) (AVONEX), an immunomodulatory cytokine, is a candidate therapeutic agent for IBM. The proposed pilot study is a six-center, randomized, placebo-controlled, parallel group study of 30 mcg of betaINF1a, administered intramuscularly once a week in 30 patients with IBM. The primary goals of the study are to examine the safety and tolerability of betaINF1a in patients with IBM, refine outcome measures of muscle function in IBM, and extend previous data on the natural history of IBM. A secondary goal is to obtain preliminary data on the efficacy of betaINF1a by measuring changes in muscle strength and mass. The study will include only patients that meet rigid criteria for IBM and who do not have complicating illness. The testing methods have been validated by the investigators in other studies of muscle disease. The information obtained in this study will be essential for the effective design and conduct of pivotal trials in IBM.

包涵体肌炎（IBM）是50岁以上成人最常见的获得性肌肉疾病。IBM有典型的临床表现，但需要肌肉活检才能确诊。尽管有令人信服的证据表明免疫异常在其发病机制中的重要性，但该疾病的病因尚不清楚，并且通常认为对常规免疫抑制剂治疗具有耐药性。β干扰素-1a (betaINF1a) （AVONEX）是一种免疫调节细胞因子，是IBM的候选治疗剂。拟议的试点研究是一项六中心，随机，安慰剂对照，平行组研究，30 mcg betaINF1a，每周肌肉注射一次，30例IBM患者。该研究的主要目标是检查betaINF1a在IBM患者中的安全性和耐受性，完善IBM患者肌肉功能的结果测量，并扩展先前关于IBM自然史的数据。第二个目标是通过测量肌肉力量和质量的变化来获得betaINF1a功效的初步数据。这项研究将只包括符合IBM严格标准且没有并发症的患者。这些测试方法已经在其他肌肉疾病的研究中得到了研究者的验证。本研究中获得的信息对于IBM关键试验的有效设计和实施至关重要。