This research study is a Phase II clinical trial. Phase II clinical trials test the
effectiveness of an investigational intervention to learn whether the intervention, in this
case, the GVAX vaccine, works in preventing your MDS, CMML, or AML from relapsing after your
allogeneic stem cell transplantation. "Investigational" means that the vaccine is still being
studied and that research doctors are trying to find out more about it-such as the side
effects it may cause, and if the vaccine is effective. It also means that the FDA has not yet
approved the vaccine for your type of cancer.
You are being asked to participate in this trial because you have advanced myelodysplastic
syndrome (MDS), Chronic Myelomonocytic Leukemia (CMML), or acute myeloid leukemia (AML). Your
doctor has determined that you are a candidate for an allogeneic stem cell transplant as
treatment for your MDS/CMML/AML. Allogeneic stem cell transplantation is a standard treatment
for MDS/CMML/AML. It can be effective because the cells from your donor (also known as the
graft) could form a new immune system that can fight against the MDS/CMML/AML cells in your
body. This is also known as the "graft-versus-leukemia" or "GVL" effect. In patients with
advanced MDS, CMML, or AML that is not in remission at the time of transplantation, as in
your case, relapse remains the number one cause of transplant failure. As such, this clinical
trial is designed to assess whether adding a leukemia vaccine early after transplantation
could stimulate donor cells to fight your cancer and improve transplant outcomes.
In recent years, researchers at the Dana-Farber Cancer Institute have discovered that GVAX, a
vaccine made from the patient's own cancer cells engineered to produce a protein called
GM-CSF, can be effective in stimulating a powerful immune response specific to that cancer.
GM-CSF is a naturally occurring hormone in the body that helps the immune system fight
infections and diseases. The GVAX vaccine is made in the laboratory by using a virus (called
adenovirus, which has been modified so it cannot cause illness) to insert the GM-CSF gene
into tumor cells. The cells are then irradiated, which prevents them from being able to grow,
before being administered to patients in a series of vaccinations.
A previous phase I clinical trial using this GVAX vaccine in patients with MDS/AML after
allogeneic transplantation demonstrated that the GVAX vaccine is safe, and the survival
outcomes were encouraging. The current randomized phase II study will investigate this
vaccine further and gather more information to assess the activity.
If you participate in this research study, you will be "randomized" to receive either GVAX
vaccination or placebo (a saline solution) vaccination. Randomization means that you are put
into a group by chance. It is like flipping a coin. There is a 50% chance that you will
receive the GVAX vaccine and a 50% chance you will receive placebo. Neither you nor your
transplant doctor(s) will know which you will be receiving.
The primary goal of this trial is to assess if there will be a difference in the percentage
of cancer free survivors in the vaccinated vs. placebo group at 18 months after transplant.
Phase:
Phase 2
Details
Lead Sponsor:
Dana-Farber Cancer Institute
Collaborators:
Beth Israel Deaconess Medical Center Massachusetts General Hospital