CC-4047 (Pomalidomide) for Graft vs. 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|
- Overall Response (Complete Response + Partial Response + Other) [ Time Frame: 1 year after last dose of CC-4047 ] [ Designated as safety issue: No ]
- CR is defined as complete resolution in all of signs and symptoms at all affected organs and tissues
- PR is defined as improvement in greater than or equal to 1 organ/tissue with no progression in any other affected organ/tissue
- Improvement in chronic GvHD symptoms less than what meets the definition of a PR is defined as other
- Progressive disease is defined as failure of therapy to control chronic GvHD despite increasing the dose of primary therapy or adding second line treatments
- No response is defined as no change in disease.
- Safety as Measured by the Most Common Adverse Effects and Reasons for Dose Reductions or Study-discontinuation [ Time Frame: 30 days after last dose of CC-4047 or until resolution of event ] [ Designated as safety issue: Yes ]-Toxicities will be graded according to the NCI CTCAE v3.0.
|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 an 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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00770757
|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|