Study on the Safety and Effectiveness of VELCADE® in the Treatment of Graft-Versus-Host Disease
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase II Trial of VELCADE® (Bortezomib) for Steroid Refractory Acute GVHD|
- Response to Bortezomib (VELCADE®) [ Time Frame: Through 30 days post-treatment ] [ Designated as safety issue: No ]
Response is the primary endpoint of this study and will be scored on day 21 (3 weeks after the first dose of VELCADE) and every 3 weeks subsequently. Patients who progress or expire before the end of the study will be considered non-responders.
Patients are evaluated for response in an organ if they have AGVHD in that organ at the start of treatment with VELCADE or if AGVHD develops after the start of VELCADE, but before the time period of evaluation. Complete response in an organ is defined as no evidence clinical or biochemical signs of AGVHD. For the overall assessment, it is defined as complete resolution of rash, abnormal LFTs, and absence of diarrhea attributed to AGVHD.
Partial response is defined as a one stage decrease in any organ system without worsening in other organ systems.
- Number of Toxicities Related to Bortezomib (VELCADE®) [ Time Frame: Through 30 days post-traeatment ] [ Designated as safety issue: Yes ]Observe the toxicities of VELCADE® when administered to recipients of allogeneic hematopoietic stem cell transplant in the setting of steroid refractory or steroid dependent acute graft-versus-host disease.
|Study Start Date:||November 2005|
|Study Completion Date:||September 2009|
|Primary Completion Date:||September 2008 (Final data collection date for primary outcome measure)|
Experimental: Bortezomib for Treatment of GHVD
To determine if bortezomib (VELCADE®) will successfully inhibit T-cell responses in clinically acute graft-versus-host disease (GVHD) after allogeneic hematopoietic stem cell transplantation (HSCT).
Bortezomib at 1.3 mg/m2/dose given twice weekly for two weeks followed by a 10-day rest period. If patients have a complete response, they will receive additional cycles of bortezomib.
Graft-versus-host disease (GVHD) is a serious complication after bone marrow transplantation from another donor. GVHD is caused by certain cells called lymphocytes. Normally these cells make immune reactions that help protect the body from foreign substances that cause infection. Here, these cells attack the normal tissues of the body as if they were foreign substances. This interferes with the normal function of vital organs and results in their damage. In GVHD these cells attack the skin, liver and bowel. GVHD also increases the chances of infection.
VELCADE® is a drug that inhibits certain immune reactions that happen when lymphocytes encounter foreign substances. We are doing this research to determine if VELCADE® may be useful in treating GVHD.
VELCADE® is approved by the Food and Drug Administration (FDA) for the treatment of multiple myeloma in patients who have received at least two prior therapies and have demonstrated disease progression on their last therapy. Its effectiveness is also being tested in other cancers. The dose of the drug being used in this research study is the same as what is used for the treatment of multiple myeloma. It has not been approved by the FDA for use in GVHD. Therefore, using VELCADE® for GVHD is experimental in this research study.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00408928
|United States, Pennsylvania|
|Thomas Jefferson University|
|Philadelphia, Pennsylvania, United States, 19107|
|Principal Investigator:||John Wagner, MD||Thomas Jefferson University|