A Phase I Study of Carfilzomib in Patients with Previously Treated Systemic Light Chain Amyloidosis

Full Title

A Phase I/II Dose-Escalation Study of Carfilzomib in Patients with Previously-Treated Systemic Light-Chain (AL) Amyloidosis


Amyloidosis is a disorder in which abnormal proteins build up in tissues and organs. Clumps of the abnormal proteins are called amyloid deposits. Amyloidosis may be treated with chemotherapy, but the disease often returns.

In this study, researchers want to find the highest dose of the drug carfilzomib that can be given safely in patients with systemic light chain amyloidosis that persists or has returned 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, resulting in cell death. Turning off the proteasome may inhibit the progression of systemic light chain amyloidosis.

Carfilzomib is already approved for treating some patients with multiple myeloma; its use in this study is considered investigational. It is given intravenously (by vein).


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

  • Patients must have systemic light chain amyloidosis that persists or has returned despite at least one regimen of prior therapy.
  • For patients who had an autologous stem cell transplant, at least 3 months must have passed between the 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 age 18 and older.

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