Rituximab Therapy in Refractory Adult and Juvenile IIM

难治性成人和青少年 IIM 的利妥昔单抗治疗

• 批准号: 8151596
• 负责人: CHARLES ODDIS
• 金额: $ 19.86万
• 依托单位: UNIVERSITY OF PITTSBURGH AT PITTSBURGH
• 依托单位国家: 美国
• 财政年份: 2004
• 资助国家: 美国
• 起止时间: 2004-09-30 至 2011-01-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8151596
• 关键词: Address; Adolescent; Adult; Adult Dermatomyositis; Adverse effects; Aftercare; Ambulatory Care Facilities; Antibodies; Autoimmune Diseases; B-Lymphocytes; Biopsy; Blood; CD20 Antigens; Childhood; Clinical Trials; Collection; Contracts; Dermatomyositis; Disease; Effectiveness; Electrocardiogram; Engineering; Enrollment; Functional disorder; Goals; Hour; Human; Immune; Immunologic Factors; Infusion procedures; Measurement; Measures; Mediating; Methods; Monitor; Monoclonal Antibodies; Mus; Muscle; Myopathy; Myositis; Neurologist; Non-Hodgkin' s Lymphoma; Outpatients; Participant; Patient Participation; Patients; Pharmaceutical Preparations; Phase; Placebos; Polymyositis; Procedures; Questionnaires; Randomized; Refractory; Rheumatoid Arthritis; Screening procedure; Surface; Systemic Lupus Erythematosus; Testing; Therapeutic; Time; Urine; Visit; advanced disease; base; cancer type; design; effective therapy; improved; muscle strength; novel therapeutics; response; rheumatologist; rituximab

Rituximab is a man-made antibody used to treat certain types of cancer. This study will determine whether rituximab is an effective treatment for adult and pediatric patients with dermatomyositis or polymyositis. Study hypotheses: 1) The time to improvement in Group A patients (receiving rituximab first) will occur significantly earlier than in Group B patients (receiving rituximab later). 2) The proportion of patients improved at Week 8 of the treatment phase will be significantly greater in Group A compared with Group B. Rituximab is a chimeric, murine-human, genetically engineered monoclonal antibody directed against the CD20 antigen found on the surface B-lymphocytes and is known to deplete B cells when administered intravenously. It is approved to treat non-Hodgkin's lymphoma. Rituximab has been used for autoimmune diseases such as systemic lupus erythematosus (SLE), rheumatoid arthritis (RA), and immune-mediated hematologic disorders. It has also been studied and used in small numbers of patients with myositis. This study will evaluate the efficacy of rituximab in treating refractory adult and pediatric patients with dermatomyositis and adult polymyositis. A patient's participation in this study will last approximately 45 weeks. At screening, participants will have a physical exam, muscle strength assessment, an electrocardiogram, and blood and urine collection; they will also be asked to complete several questionnaires. All participants will receive 2 infusions of rituximab and 2 infusions of placebo. Participants will be randomly assigned to one of two groups. Group A will receive rituximab at Weeks 0 and 1 and placebo at Weeks 8 and 9. Group B will receive placebo at Weeks 0 and 1 and rituximab at Weeks 8 and 9. Each infusion will be given on an outpatient basis over a minimum of approximately 5 hours' time. There will be a total of 14 study visits. All participants will visit the outpatient clinic at selected time points for muscle strength testing, a physical exam, disease activity measurements, and blood collection. During the study, participants will be monitored closely for improvement or worsening of their disease and for serious drug related side effects. Some participants will be asked if they are willing to undergo 2 muscle biopsy procedures, 1 prior to receiving study medication and 1 after receiving study medication, to determine the effects of rituximab on muscle tissue.

利妥昔单抗是一种用于治疗某些类型癌症的人造抗体。这项研究将确定利妥昔单抗是否是成人和小儿皮肌炎或多发性肌炎患者的有效治疗方法。研究假设：1）A组患者改善（首先接受利妥昔单抗）的时间要比B组患者早（稍后接受利妥昔单抗）的时间要早​​得多。 2）与B组相比，A组的第8周在治疗阶段的第8周将明显更大的患者比例的比例是一种嵌合，鼠 - 人类，基因工程性的单克隆抗体，针对在表面B淋巴细胞上发现的CD20抗原，并已知会在施用的insplete insplete Bymphocytes上消耗掉静脉内。它被批准用于治疗非霍奇金的淋巴瘤。利妥昔单抗已用于自身免疫性疾病，例如全身性红斑狼疮（SLE），类风湿关节炎（RA）和免疫介导的血液学疾病。它也已被研究并用于少数肌炎患者。这项研究将评估利妥昔单抗在治疗耐火性成人和小儿皮肌炎和成人多发性肌炎患者方面的疗效。患者参与这项研究将持续大约45周。在筛查时，参与者将进行体格检查，肌肉力量评估，心电图以及血液和尿液的收集；还将要求他们填写几份问卷。所有参与者将收到2次输注的利妥昔单抗和2次输注安慰剂。参与者将被随机分配给两个组之一。 A组将在第0周和第1周接受利妥昔单抗，并在第8周和第9周接受安慰剂。第B组将在第0和第1周接受安慰剂，在第8和第9周将在第8周和第9周接受Rituximab。每次输注将在至少5个小时的时间内在门诊中进行门诊。总共将有14次学习访问。所有参与者将在选定的时间点参观门诊诊所，进行肌肉力量测试，体格检查，疾病活动测量和血液收集。在研究期间，将密切监视参与者，以改善或恶化其疾病以及严重的药物相关副作用。将询问一些参与者是否愿意接受2种肌肉活检手术，1个在接受学习药物之前的1例和1次接受学习药物后，以确定利妥昔单抗对肌肉组织的影响。