A Randomized, Double Blind, Placebo Controlled Pilot Trial of Rituximab for Non-splenectomized Adults With Acute Immune Thrombocytopenic Purpura Receiving Standard Treatment (R-ITP)
Overview
- Phase
- Phase 2
- Intervention
- Rituximab
- Conditions
- Purpura, Thrombocytopenic, Idiopathic
- Sponsor
- Hamilton Health Sciences Corporation
- Enrollment
- 60
- Locations
- 7
- Primary Endpoint
- rescue therapy
- Status
- Completed
- Last Updated
- 13 years ago
Overview
Brief Summary
The purpose of this study is to assess the feasibility of a randomized, double blind, placebo controlled trial of add-on rituximab for non-splenectomized adults with acute immune thrombocytopenic purpura (ITP).
Detailed Description
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.
Investigators
Donald Arnold
MD
McMaster University
Eligibility Criteria
Inclusion Criteria
- •Non-splenectomized patients with acute ITP, where "acute ITP" is defined as a platelet count below 30 at the time that standard treatment was recommended by a physician and for which no treatment had been received for the preceding 30 days.
- •Must be receiving standard ITP treatment.
Exclusion Criteria
- •Cardiac arrhythmia.
- •Uncontrolled hypertension or inability to hold antihypertensive medications for 12 hours prior to and throughout study drug infusions.
- •Known coronary artery disease, angina pectoris or myocardial infarction within the last year.
- •Significant pulmonary disease within the last year.
- •Stroke, transient ischemic attack or venous thrombosis within the last year.
- •Secondary causes of thrombocytopenia (splenomegaly \[palpable spleen or radiologically confirmed \>14 cm\], drug-induced thrombocytopenia, hereditary thrombocytopenia, microangiopathic hemolytic anemia, myelodysplastic syndrome).
- •Chronic lymphocytic leukemia or lymphoma.
- •Active or metastatic cancer.
- •History of hepatitis B or C or HIV.
- •Active infection in the 4 weeks before randomization.
Arms & Interventions
A
Rituximab
Intervention: Rituximab
B
Saline placebo iv infusion
Intervention: Placebo
Outcomes
Primary Outcomes
rescue therapy
Time Frame: 6 months
Event free survival in controls
Time Frame: 6 months
Bleeding
Time Frame: 6 months
Feasibility of recruitment
Time Frame: 3 years
Degree of adherence to the study protocol
Time Frame: 3 years
Secondary Outcomes
- Platelet count response(6 months)
- Quality of life(6 months)
- Circulating CD-20 positive lymphocytes(6 months)
- Platelet associated IgG(6 months)