NCT02649504

Rituximab and Dexamethasone Followed by Mycophenolate Mofetil or Placebo in Patients With Immune Thrombocytopenia

The Study of Immunotherapy With Rituximab and Pulse Dexamethasone Followed by With Mycophenolate Mofetil or Placebo in Adult Patients With Persistent and Chronic Immune Thrombocytopenia

• Status: Withdrawn
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Weill Medical College of Cornell University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The investigators will attempt to further increase the cure rate of ITP with medical therapy by providing maintenance therapy with Mycophenolate mofetil (MMF) to persistent/chronic ITP patients after treating them with induction therapy combining rituximab and dexamethasone. The investigators will randomly assign patients to MMF versus placebo in order to demonstrate safety (e.g., for infectious risk) and efficacy (platelet counts stably \>50x109/L more than 1 year off therapy).

Detailed description

There are significant numbers of Immune Thrombocytopenia (ITP) patients 18 years of age or older who relapse and become refractory after short responses to initial therapy (usually corticosteroids) or who do not respond at all. Despite a variety of available therapies, there are patients with persistently low platelet counts and bleeding complications, and there are other patients who suffer from the side effects of current treatments. Thus, there still remains an unmet need for better treatments in these patients. Based on the investigators' experience, it is expected that most of ITP patients treated with intensive induction therapy (Dexamethasone combined with rituximab, R+3D) will increase their platelet counts, and the investigators hope that a further 6-month course of Mycophenolate Mofetil will help patients to maintain continuous response and even achieve a cure of ITP. The increase in platelet count will likely result in a decreased risk for bleeding and better health-related quality of life. In addition, responders potentially will be able to stop concomitant ITP medications and will not suffer from adverse events of various ITP therapies and will avoid undergoing splenectomy. This multi-center, randomized study will help physicians determine the best treatment option for ITP patients and may help to establish a new standard of care. The knowledge to be gained is that of: 1. whether R+3D and placebo confirms the previous data on R+3D 2. whether R+3D + MMF increases the cure rate of ITP 3. whether R+3D + MMF increases the risk of serious infections The investigators do not believe that the triple therapy will result in a high rate of serious infections based on the past track record of R+3D and of MMF in combination with other stronger immunosuppressive medications such as cyclosporine and tacrolimus. Therefore the risk will be limited and worth the increase in cure rate that we expect.

Conditions

• Immune Thrombocytopenia

Interventions

Type	Name	Description
DRUG	Rituximab	Rituximab will be given at the standard dose of 375mg/m2 on days 1, 8, 15, 22 of the study
DRUG	Dexamethasone	Dexamethasone will be given at dose 40 mg/day on days 1-4, 15-18, and 29-32 (+/- 3 days) of the study.
DRUG	Mycophenolate mofetil	Patients allocated to the active drug arm will be given Mycophenolate mofetil starting on day 33, 500 mg twice daily for the first week and then escalation to 1000 mg twice daily for 24 weeks.
DRUG	Placebo	Patients allocated to the placebo arm will be given placebo starting on day 33, 500 mg twice daily for the first week and then escalation to 1000 mg twice daily for 24 weeks.

Timeline

• Start date: 2016-12-01
• Primary completion: 2016-12-01
• First posted: 2016-01-07
• Last updated: 2018-08-14

Regulatory

• FDA-regulated drug study