A Study of Captisol-Enabled Melphalan in Patients with Multiple Myeloma and Light Chain Amyloidosis Receiving an Autologous Stem Cell Transplant


Full Title

Pharmacokinetic (PK)-directed Dosing of Captisol Enabled Melphalan for Patients with Multiple Myeloma or Light Chain (AL) Amyloidosis Undergoing High Dose Therapy and Autologous Hematopoietic Progenitor Cell Transplant


Multiple myeloma is a cancer of the bone marrow. Specifically, the cancer is due to a type of bone marrow cell called a plasma cell. Myeloma plasma cells typically can destroy bones, hurt the kidneys, and cause immune problems. Amyloidosis is a similar cancer of bone marrow plasma cells. With amyloidosis, however, the plasma cells make abnormal, misfolded proteins that build up in tissues and organs, preventing them from working properly. In amyloidosis, often the heart, kidneys, gastrointestinal tract, liver, and nervous systems are involved.

Using a patient’s own cells (special cells called “stem cells”) to help increase blood counts after high doses of chemotherapy is called autologous stem cell transplantation (ASCT). ASCT is a treatment approach used for patients with multiple myeloma and light chain amyloidosis.

Melphalan is a drug used to treat multiple myeloma and light chain amyloidosis. Captisol-enabled melphalan is a new form of the drug which is more stable and may cause fewer side effects than melphalan. Because captisol-enabled melphalan is a more stable form of melphalan, its levels can be measured in the blood. In this study, researchers are seeking to learn if they can achieve the safest and most effective dose of captisol-enabled melphalan that can be given in patients with multiple myeloma and light chain amyloidosis who are receiving high-dose chemotherapy and will be undergoing an autologous stem cell transplant. It is hoped that measuring blood levels of captisol-enabled melphalan will allow researchers to individualize patient doses, which can make stem cell transplantation safer and more effective.


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

  • Patients must have symptomatic multiple myeloma or light chain amyloidosis and be candidates for high-dose chemotherapy and autologous stem cell transplantation.
  • This study is for patients between the ages of 18 and 75.

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