A Randomized, Double Blind, Placebo Controlled Pilot Trial of Rituximab for Non-splenectomized Adults With Acute Immune Thrombocytopenic Purpura Receiving Standard Treatment (R-ITP)
Immune thrombocytopenic purpura (ITP) is an autoimmune disease characterized by severe
thrombocytopenia and bleeding. With current standard therapies, adult-onset ITP tends to
recur thus exposing patients to prolonged risks of hemorrhage and toxicities of standard
treatments. Rituximab, a chimeric anti-CD20 monoclonal antibody, has been shown to be
effectively raise the platelet count in some patients with ITP and there is clinical and
biological evidence to suggest that, if given early, rituximab may prevent ITP relapses.
We have designed a randomized, double blind, placebo controlled pilot trial of rituximab for
the treatment of non-splenectomized adults with acute ITP who are receiving standard
treatments. The primary objectives of this trial are to determine the feasibility of
recruitment, randomization and blinding; the safety of rituximab in ITP; and the event rate
in the control group which will be used to calculate the sample size for a larger trial.
Secondary objectives are to determine rates of 6-month event free survival where an event is
defined as any of: a platelet count <50; the need for rescue treatment; or significant
bleeding. Data from this pilot trial will inform the design of a larger phase III trial.
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Prevention
Feasibility of recruitment
Donald M Arnold, MD
Canada: Health Canada