A Phase I/IIA Study of Carfilzomib plus High-Dose Melphalan Before Autologous Stem Cell Transplant in Patients with Multiple Myeloma

Full Title

Phase 1/2a Study of Carfilzomib + High Dose Melphalan as Preparative Regimen for Autologous Hematopoietic Stem Cell Transplantation in Multiple Myeloma


Autologous stem cell transplantation — removing some of a patient’s own stem cells before chemotherapy and returning them afterward, to re-establish a new immune and blood-forming system — is a therapy for multiple myeloma. High doses of the chemotherapy drug melphalan are traditionally used for this treatment, but in many patients the myeloma returns.

The purpose of this study is to see if giving the drug carfilzomib in combination with melphalan is a safe and effective preparation for autologous stem cell transplantation in patients with multiple myeloma that has returned or persists despite standard treatment. Doctors hope that adding carfilzomib will reduce the risk of myeloma recurrence more than melphalan alone. Carfilzomib is approved for treating myeloma that persists despite prior therapy.

Carfilzomib is a proteasome inhibitor. The proteasome breaks down proteins that are no longer useful to a cell. Proteasome inhibitors turn off the proteasome, causing the proteins to build up in the cell and resulting in cell death. Myeloma cells require a lot of proteins and an active proteasome to survive, so drugs that turn off the proteasome may inhibit myeloma growth.


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have multiple myeloma that has returned following or has stopped responding to standard treatment.
  • For patients who had prior autologous stem cell transplantation, at least a year must have passed since the last transplant and entry into the study.
  • Patients must be able to be ambulatory for more than half of their normal waking hours.
  • This study is open to patients ages 18 to 70.

For more information about this study and to inquire about eligibility, please contact Dr. Heather Landau at 212-639-8808.