NCT01672229

Bortezomib in Patients With Chronic Graft Versus Host Disease

A Pilot Study Of Weekly Subcutaneous Bortezomib In Patients With Steroid-Refractory Or -Dependent Chronic Graft Versus Host Disease

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 16 (actual)
• Sponsor: Mehrdad Abedi, MD · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This study will investigate whether bortezomib can control the immune system and can be used to treat GVHD. Bortezomib has been used with not too many serious side effects in patients with multiple myeloma who will undergo transplant and also for acute graft versus host disease.

Detailed description

Bone marrow transplantation offers great promise for the treatment of a variety of diseases, particularly hematological malignancies. The incidence of acute GVHD has significantly decreased due to significant improvements in human leukocyte antigen (HLA) matching of the donors and recipients, more efficient GVHD prophylaxis regimens and the use of reduced-intensity preparative regimen. However, cGVHD remains a significant cause for increased morbidity and mortality associated with allogeneic stem cell transplantation. While many of the patients with cGVHD respond initially to higher doses of steroids, cGVHD usually relapses during or following steroid taper. Because of the significant impact of steroids on this patient population, there is an urgent need for medications to take the place of high dose steroid use in this patient population. We hypothesize that bortezomib can modulate the immune system and can be used to treat GVHD. At the same time bortezomib post transplant can induce a graft versus leukemia or lymphoma effect. Bortezomib has been used with minimal toxicity in post transplant setting for patients with aggressive multiple myeloma and also for acute graft versus host disease.

Conditions

• Graft Versus Host Disease

Interventions

Type	Name	Description
DRUG	Bortezomib	If improvements are seen at any dose level and patients have no DLTs, they will stay at their dose level until the end of the study. This is to avoid any possible toxicity while the patient is benefiting from their current dosing of bortezomib. If the continuous improvement in GVHD stalls at any point, or the GVHD progresses after the original improvement, while the dose level is maintained, then the dose will be increased to the next dose level. Patients will remain enrolled until exacerbation of the GVHD on increasing dose schedule or closure of the study. Clinical activity will be monitored every other week after the initiation of bortezomib until the study closes.

Timeline

• Start date: 2012-07-01
• Primary completion: 2018-04-09
• Completion: 2018-04-09
• First posted: 2012-08-24
• Last updated: 2020-05-28

Locations

1 site across 1 country: United States