NCT01605032

Busulfan, Melphalan, and Bortezomib Before First-Line Stem Cell Transplant in Treating Patients With Multiple Myeloma

Phase II Study Assessing the Efficacy and Toxicity of PK--directed Intravenous Busulfan in Combination With High--Dose Melphalan and Bortezomib as Conditioning Regimen for First--Line Autologous Hematopoietic Stem Cell Transplantation in Patients With Multiple Myeloma

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 19 (actual)
• Sponsor: Albert Einstein College of Medicine · Academic / Other
• Sex: All
• Age: 18 Years – 72 Years
• Healthy volunteers: Not accepted

Summary

This phase II trial studies how well busulfan, melphalan, and bortezomib before first-line stem cell transplant works in treating patients with multiple myeloma. Giving chemotherapy before a peripheral blood stem cell transplant may stop the growth of cancer cells by stopping them from dividing or killing them. After treatment, stem cells are collected from the patient's blood and stored. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy.

Detailed description

PRIMARY OBJECTIVES: I. To determine the complete response rate as defined by the International Myeloma Working Group (IMWG) criteria for patients with multiple myeloma treated with high dose chemotherapy with pharmacokinetic (PK) directed intravenous (IV) busulfan, bortezomib and melphalan (Bu/BTZ/Mel140) followed by autologous hematopoietic stem cell transplantation (ASCT) as first line therapy. SECONDARY OBJECTIVES: I. To determine the overall response rate of the regimen Bu/BTZ/Mel140. II. To determine the treatment related toxicity and mortality of the regimen, including 100-day mortality rates. III. To determine the duration of response, time to progression, progression-free survival, event-free survival and overall survival for this conditioning regimen. IV. To determine whether there is a gender or race difference in the pharmacokinetic profile of IV busulfan. V. To determine methylation and gene expression signatures of pre-treatment bone marrow plasma cells and explore associations of these signatures with outcome. OUTLINE: CONDITIONING: Patients receive busulfan IV over 3 hours on days -6 to -3, melphalan IV over 20 minutes on day -2, and bortezomib IV over 3-5 seconds on days -6, -3, 1, and 4. TRANSPLANT: Patients undergo autologous peripheral blood stem cell transplant (PBSCT) on day 0. After completion of study treatment, patients are followed up for up to 5 years.

Conditions

• DS Stage I Plasma Cell Myeloma
• DS Stage II Plasma Cell Myeloma
• DS Stage III Plasma Cell Myeloma
• Refractory Plasma Cell Myeloma

Interventions

Type	Name	Description
DRUG	Busulfan	Given IV
DRUG	Melphalan	Given IV
DRUG	Bortezomib	Given IV
PROCEDURE	Autologous Hematopoietic Stem Cell Transplantation	Undergo autologous PBSCT
PROCEDURE	Peripheral Blood Stem Cell Transplantation	Undergo autologous PBSCT

Timeline

• Start date: 2012-02-01
• Primary completion: 2018-03-01
• Completion: 2018-03-01
• First posted: 2012-05-24
• Last updated: 2020-08-20
• Results posted: 2020-08-20

Locations

4 sites across 1 country: United States