NCT00437086

Bortezomib in Treating Patients With Advanced Myeloproliferative Disorders

A Prospective Open-Label Pilot Trial of PS-341 (Bortezomib; VELCADE) for the Therapy of Symptomatic Advanced Myeloproliferative Disorders

• Status: Completed
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: Mayo Clinic · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

RATIONALE: Bortezomib may stop the growth of abnormal cells by blocking some of the enzymes needed for cell growth and by blocking blood flow to the abnormal cells. PURPOSE: This clinical trial is studying the side effects and how well bortezomib works in treating patients with advanced myeloproliferative disorders.

Detailed description

OBJECTIVES: Primary * Determine the efficacy of bortezomib in patients with symptomatic advanced myeloproliferative disorders (i.e., myelofibrosis with myeloid metaplasia, chronic myelomonocytic leukemia, or FIP1LI-PDGFRA-negative mast cell disease). * Determine the safety of this drug when administered on a modified schedule in these patients. Secondary * Determine the effect of this drug on bone marrow cellularity, tryptase-positive mast cells, reticulin fibrosis, osteosclerosis, and angiogenesis in responding patients OUTLINE: This is a prospective, open-label, pilot, multicenter study. Patients are stratified according to disease (systemic mast cell disease vs chronic myelomonocytic leukemia vs myelofibrosis with myeloid metaplasia). Patients receive bortezomib IV weekly for 4 weeks. Treatment repeats every 5 weeks for up to 2 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a response (complete remission, partial remission, or minimal remission) after 2 courses may receive an additional 6 courses of therapy. Patients who achieve stable disease with acceptable toxicities after 2 courses receive bortezomib IV at a higher dose twice weekly for 2 weeks. Treatment with a higher dose of bortezomib repeats every 3 weeks for up to 6 courses. Patients who are responders undergo bone marrow aspirate or biopsy and peripheral blood collection for evaluation of bone marrow cellularity, tryptase-positive mast cells, reticulin fibrosis, osteosclerosis, and angiogenesis by fluorescent in situ hybridization (FISH), immunohistochemistry, and other immunological laboratory methods. After completion of study therapy, patients are followed periodically for up to 3 years. PROJECTED ACCRUAL: A total of 30 patients will be accrued for this study.

Conditions

• Chronic Myeloproliferative Disorders
• Leukemia

Interventions

Type	Name	Description
DRUG	PS-341	1.6 mg/m2 by IV; 4 out of 5 weeks

Timeline

• Start date: 2005-09-01
• Primary completion: 2008-11-01
• Completion: 2008-11-01
• First posted: 2007-02-19
• Last updated: 2014-10-17

Locations

3 sites across 1 country: United States