CC-4047 (Pomalidomide) for Graft vs. Host Disease
This study will test the safety and effectiveness CC-4047 (pomalidomide) in patients with advanced, steroid refractory graft-versus-host disease.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase 2, Open-Label, Single-Arm, Pilot Study of Safety and Efficacy of CC-4047 (Pomalidomide) in Patients With Advanced Chronic Graft-Versus-Host Disease Developing After Allogeneic Hematological Stem Cell Transplantation|
- Efficacy of CC-4047 [ Time Frame: 1 year after last dose of CC-4047 ] [ Designated as safety issue: No ]Overall response (complete response, partial response based on NIH Consensus Response Criteria Working Group)
- Safety of CC-4047 [ Time Frame: 30 days after last dose of CC-4047 or until resolution of event ] [ Designated as safety issue: Yes ]Safety (type, frequency, severity, and relationship to CC-4047)
|Study Start Date:||February 2009|
|Study Completion Date:||October 2011|
|Primary Completion Date:||October 2010 (Final data collection date for primary outcome measure)|
Experimental: CC-4047 Arm
CC-4047 2 mg orally every day for 1 course (12 weeks or 84 days). Every 28 days of treatment are considered as 1 cycle and every 3 cycles are are considered as 1 course of treatment. A total of 4 courses of treatment are planned (12 months).
Other Name: Pomalidomide
Chronic Graft vs. Host Disease is a major complication after allogeneic hematopoietic stem cell transplantation developing in 30 - 70% of patients. It is a multisystem alloimmune and autoimmune disorder with a negative impact on quality of life and functional status, increased need for extended immunosuppression and is the leading cause of late transplant related mortality.
CC-4047 is a novel immune modulatory drug that is a thalidomide analog with a 4,000 fold greater inhibition of TNF-α production related to thalidomide. Several features of CC-4047 suggest that this drug may be useful in treating chronic GVHD including in vitro suppression of TNF-α production, increasing Th1 and stimulation of IL-12 and sIL-Rα.
This study is a phase 2, open-label, single-arm, pilot study of efficacy and safety of CC-4047 in patients with advanced chronic GvHD who failed to achieve a response with high-dose corticosteroids or second line systemic immunosuppressive therapy.
|United States, Missouri|
|Washington University School of Medicine|
|St. Louis, Missouri, United States, 63122|
|Principal Investigator:||John F. DiPersio, M.D., Ph.D.||Washington Univerisity School of Medicine|