This phase 2 clinical trial will test the effectiveness of a combination of panobinostat (Farydak), gemcitabine (Gemzar), busulfan (Busulfex), and melphalan (Alkeran), and a stem cell transplant in treating multiple myeloma. The primary outcome will be measured by the time until the disease progresses. This trial is being conducted in Houston, Texas.
High-dose chemotherapy with autologous stem cell transplantation (where patients’ blood stem cells are collected and transplanted back after chemotherapy) is typically given as a treatment for patients with multiple myeloma. The purpose is to kill the cancer cells and replace the healthy blood forming stem cells that were damaged during chemotherapy. This method of treatment is normally effective in treating myeloma, but only a single chemotherapy drug is normally used for this therapy. A combination therapy containing panobinostat, gemcitabine, busulfan, and melphalan (all chemotherapies that kill immune cells by damaging DNA) as a first-line treatment, rather than just one agent, followed by autologous stem cell transplantation may further improve the treatment response rate.
The study will examine the time until the disease progresses for 1 year following treatment with the combination of chemotherapies.
Who are they looking for?
This study is recruiting 80 patients with relapsed or refractory multiple myeloma. Patients should have received at least 2 cycles of lenalidomide, bortezomib, or thalidomide. Participants should have adequate liver, kidney, heart, and lung function.
Participants should not have received radiotherapy to the head, neck, chest region, or abdomenal area within 1 month of enrollment. Ideal candidates should not have received autologous stem cell transplantation within 6 months prior to entry, anticancer therapy within 2 weeks of enrollment, or undergone major surgery within 4 weeks prior to entry. Those with a history of other malignancies should be disease free for 5 years.
How will it work
Patients will be divided into either a refractory disease group or a relapsed disease group. Both groups will receive the same treatment. Busulfan will be given intravenously (through IV) on days -10 and -8 to -5 of the trial (before stem cell transplantation). Palifermin (Kepivance) will be delivered intravenously on days -14 to -12 and on days 0 to 2 to help prevent inflammation of the digestive system. Patients will receive intravenous dexamethasone 2 times a day on days -9 to -2. Panobinostat will be taken daily by mouth on days -8 to -2. Gemcitabine will be given intravenously on days -8 and -3. Melphalan will be given intravenously on days -3 and -2. Stem cell transplantation will occur on day 0 of the trial.
Patients will be followed at 1 month, 100 days, 6 months, 1 year, and then every 3 to 6 months for at least 2 years. Follow-up will include bone marrow biopsy at 100 days, then once per year, as well as x-rays of all the bones in the body.