CLINICAL TRIAL: RITUXIMAB IN THE TREATMENT OF REFRACTORY ADULT AND JUVENILE DERM

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目及 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 拟议研究的主要目的是研究在IIM疾病患者中，利妥昔单抗是否会1）改善肌肉力量和2）改善疾病症状。 这是一项多中心、双盲、随机安慰剂相设计（RPPD）研究，旨在比较两组利妥昔单抗治疗患者之间达到改善定义的时间。 在确定合格性的初始筛选后，受试者将以1：1的比例随机分配至A组或B组。 A组：受试者将在第0周和第1周接受利妥昔单抗静脉输注，随后在第8周和第9周接受安慰剂输注。 B组：受试者将在第0周和第1周接受安慰剂输注，随后在第8周和第9周接受利妥昔单抗静脉输注。 入组患者将包括5岁及以上的男性和女性、成人和儿童患者，确诊或可能诊断为PM或DM（使用Bohan和Peter标准），病程超过6个月。 大约20家成人中心和18家儿科中心将在2年招募期内招募受试者。 随机化受试者总数为202例，包括76例成人PM、76例成人DM和50例儿童JDM患者。 将在斯坦福大学和LPCH筛选约10例成人和青少年受试者，随机分配6-8例受试者。 受试者将完成健康评估和总体疾病评估问卷，并接受手动肌肉测试，以评估44周和14次研究访视期间的肌无力水平。 获得改善然后随后恶化的患者可能有资格参加再治疗阶段，在那里他们将接受两次利妥昔单抗输注，并再随访44周。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The primary objective of the proposed study is to investigate whether, in patients with IIM disease; rituximab will 1) improve muscle strength and 2) improve symptoms of disease. This is a multicenter, double blind, randomized placebo phase design (RPPD) study to compare the time to achieve the definition of improvement between two groups of rituximab treated patients. After an initial screening to determine eligibility subjects will be randomized in a 1:1 fashion to either Group A or Group B. Group A: Subjects will receive rituximab intravenous infusion at weeks 0 and 1 followed by placebo infusion at weeks 8 and 9. Group B: Subjects will receive placebo infusion at weeks 0 and 1 followed by rituximab intravenous infusion at weeks 8 and 9. Enrolled patients will include male and female, adult and pediatric patients five years of age and over with a definite or probable diagnosis PM or DM (using the criteria of Bohan and Peter) with a disease duration greater than six months. Approximately 20 adult and 18 pediatric centers will enroll subjects over a 2 year recruitment period. The total number of randomized subjects will be 202, to include 76 adult PM, 76 adult DM and 50 pediatric JDM patients. Approximately 10 adult and juvenile subjects will be screened at Stanford and LPCH to randomize 6-8 participants. Subjects will complete health assessment and global disease assessment questionnaires and undergo manual muscle testing to evaluate the level of their muscle weakness over 44 weeks and 14 study visits. Patients who achieve improvement then subsequently worsen may be eligible for enrollment in a re-treatment phase where they would receive two infusions of rituximab and be followed an additional 44 weeks.