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

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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.