CC-4047 (Pomalidomide) for Graft vs. Host Disease
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|ClinicalTrials.gov Identifier: NCT00770757|
Recruitment Status : Completed
First Posted : October 10, 2008
Results First Posted : February 25, 2016
Last Update Posted : February 25, 2016
|Condition or disease||Intervention/treatment||Phase|
|Graft vs Host Disease||Drug: CC-4047||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||13 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Study Start Date :||February 2009|
|Primary Completion Date :||October 2010|
|Study Completion Date :||October 2011|
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
- Overall Response (Complete Response + Partial Response + Other) [ Time Frame: 1 year after last dose of CC-4047 ]
- 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 ]-Toxicities will be graded according to the NCI CTCAE v3.0.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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|