Lenalidomide and High-Dose Melphalan

The Study Drugs:

Melphalan is designed to damage the DNA (the genetic material of cells) of cells, which may cause cancer cells to die. High-dose melphalan is considered the standard of care for multiple myeloma.

Lenalidomide is designed to block a protein that plays a role in cell function and growth, which may cause cancer cells to die.

Study Dose Levels:

If you agree to take part in this study, you will be assigned to a dose level of lenalidomide based on when you join this study. Up to 4 dose levels of lenalidomide will be tested for safety. The first group of participants will receive the lowest dose level. Each new group will receive a higher dose than the group before it, if no intolerable side effects were seen.

All participants will receive the same dose level of melphalan.

Once the highest tolerable dose of the combination of melphalan and lenalidomide is found, the next group of patients will be randomly assigned to 1 of 4 possible groups that will be determined by the computer, based on the safest and most effective dose level at that particular point.

Study Drug Administration:

You will take lenalidomide by mouth 1 time a day beginning 8 days before the stem cell transplant (Day -8). You will take the drug for 7 days (Days -8 through -2). You should take it with a few sips of water.

On Days -3 and -2, you will receive melphalan by vein over 30 minutes.

On Day 0, after you have received the chemotherapy study drugs, you will receive an infusion of stem cells, which were previously collected from you. This infusion of stem cells is given in an effort to help increase blood production and strengthen your immune system. You will receive antibiotics in an effort to decrease the likelihood that you will develop an infection.

Hospitalization following transplant usually lasts about 2-4 weeks, but may be longer. Some participants may be discharged earlier and followed in the outpatient clinic.

Study Visits:

About 1 month, 3 months, and 6 months after the transplant:

• You will have a physical exam and your medical history will be recorded.
• Blood (about 2 tablespoons) and urine will be collected for routine tests and to check the status of the disease.
• About 3 months after the transplant, you will have a bone marrow biopsy and aspiration to check the status of the disease. This will be repeated more often, if your doctor thinks it is needed.

About 1 year after the transplant:

• You will have a physical exam and your medical history will be recorded.
• Blood (about 2 tablespoons) and urine will be collected for routine tests.
• You will have a bone marrow biopsy and aspiration to check the status of the disease.
• You will have x-rays of your bones to check the status of the disease.

Length of Study:

Your participation in this study will be over after the 1 year transplant follow-up visit.

If intolerable side effects from the chemotherapy occur or there is sign of disease after the transplant, you will be taken off study. If you have intolerable side effects after you receive melphalan, then you will still have the transplant. However, if intolerable side effects develop before you take melphalan, you may be taken off study without having the transplant. If you are taken off study early, you still may need to return for routine post-transplant follow-up visits, if your transplant physician decides it is necessary.

It may be life-threatening to leave the study early during the conditioning regimen without following up with the stem cell transplant, because your blood cell counts may be dangerously low.

This is an investigational study. Lenalidomide and melphalan are commercially available and FDA approved for the treatment of myeloma. However, the use of lenalidomide with melphalan before an autologous stem cell transplant is investigational.

Up to 60 patients will take part in this study. All will be enrolled at MD Anderson.