NCT03303950

Allogeneic Stem Cell Transplantation for Multiple Myeloma and Myelofibrosis

Busulfan, Fludarabine, Donor Stem Cell Transplant, and Cyclophosphamide in Treating Participants With Multiple Myeloma or Myelofibrosis

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: University of Utah · Academic / Other
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

This phase II trial studies how well busulfan, fludarabine, donor stem cell transplant, and cyclophosphamide in treating participants with multiple myeloma or myelofibrosis. Drugs used in chemotherapy, such as busulfan, fludarabine, and cyclophosphamide, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving chemotherapy before a donor stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. When the healthy stem cells from a donor are infused into the participant they may help the participant's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Giving busulfan and fludarabine before and cyclophosphamide after donor stem cell may work better in treating participants with multiple myeloma or myelofibrosis.

Detailed description

PRIMARY OBJECTIVES: I. To evaluate non-relapse mortality (NRM) up to day +100. SECONDARY OBJECTIVES: I. To evaluate non-relapse mortality (NRM) up to day +365. II. To evaluate the incidence of acute graft versus host disease (GVHD) and chronic GVHD up to day +365 post-transplant. III. To evaluate the overall survival and disease free survival up to 1 year. IV. To evaluate clinical response and molecular response (complete response and partial response) up to 1 year. OUTLINE: Participants receive busulfan intravenously (IV) over 2 hours and fludarabine IV over 30 minutes on days -5 to -2. Participants undergo hematopoietic cell transplantation (HSCT) on day 0. Participants then receive cyclophosphamide IV over 60 minutes on days 3 and 4. After completion of study treatment, participants are followed up for 1 year.

Conditions

• Anemia
• ASXL1 Gene Mutation
• EZH2 Gene Mutation
• IDH1 Gene Mutation
• IDH2 Gene Mutation
• Plasma Cell Myeloma
• Primary Myelofibrosis
• Recurrent Plasma Cell Myeloma
• Secondary Myelofibrosis
• Thrombocytopenia

Interventions

Type	Name	Description
DRUG	Busulfan	Given IV
DRUG	Cyclophosphamide	Given IV
DRUG	Fludarabine	Given IV
PROCEDURE	Hematopoietic Cell Transplantation	Undergo HSCT
OTHER	Laboratory Biomarker Analysis	Correlative studies

Timeline

• Start date: 2018-03-30
• Primary completion: 2019-05-14
• Completion: 2020-02-19
• First posted: 2017-10-06
• Last updated: 2022-05-05
• Results posted: 2020-05-27

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study