Phase 2 Study of Bortezomib (Velcade) for the Treatment of Steroid Refractory Chronic Graft-vs-Host Disease
Graft vs. host disease represents the most formidable problem following allogeneic
hematopoietic stem cell transplantation for hematological diseases. Chronic
graft-versus-host disease (cGVHD) is the most serious and common long-term complication of
this treatment in patients surviving more than 100 days. Some patients are requiring
immunosuppressive therapy for years. cGVHD is associated with a high degree of morbidity
and mortality and remains a major cause of late death. When standard treatment with
steroids is ineffective or poorly tolerated, effective therapeutic options are limited.
Patients with uncontrolled cGVHD with initial therapy including systemic steroids respond
poorly to second line options. Recent studies have demonstrated Bortezomib safety in the
prophylaxis, and improvement of GVHD suggesting its efficacy.This study is done to evaluate
the potential of bortezomib in treating refractory cGVHD.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
To evaluate the effectiveness and safety of the proteasome inhibitor Bortezomib (Velcade) in the treatment of steroid refractory chronic Graft-vs-Host Disease (cGVHD)
Total time frame including treatment and follow up is 19 months(Maximum of 6 cycles during initial treatment phase, 6 cycles in maintenance phase, and follow up phase for 6 months). Subjects will be assessed for signs and symptoms of cGVHD on day 1 of every cycle using standardized criteria for diagnosis of cGVHD, new clinical scoring system, and new guidelines for global assessment of chronic GVHD severity per NIH consensus development project on criteria for clinical trials in chronic GVHD. Safety will be assessed in terms of adverse events for 30 days following the last dose to a maximum of 6 months. Adverse events will be recorded through out the trial and will be followed by investigator until resolution.
Alan Miller, MD, PhD
Charles A. Caner Center, Baylor University Medical Center
United States: Food and Drug Administration
|Charles A. Cancer Center, Baylor University Medical Center||Dallas, Texas 75246|