GVAX vs. Placebo for MDS/AML After Allo HSCT

Status: Recruiting
Phase: Phase 2
Diagnosis: Hematopoietic Stem Cell Transplant
NCT ID: NCT01773395 (View complete trial on ClinicalTrials.gov)
DFCI Protocol ID: 12-217


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 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 hte 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) 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/AML. Allogeneic stem cell transplantation is a standard treatment for MDS/AML. It can be effective because the cells from your donor (also known as the graft) could from a new immune system that can fight against the MDS/AML cells in your body. This is also known as the "graft-versus-leukemia" or "GVL" effect. In patients with advanced MDS 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 enrollment.


Conducting Institutions:
Dana-Farber Cancer Institute, Brigham and Women's Hospital

Overall PI:
Vincent Ho, MD, Dana-Farber Cancer Institute

Site-responsible Investigators:

Dana-Farber Cancer Institute: Mildred Pasek, mpasek@partners.org

Eligibility Criteria

Inclusion Criteria: - AML, MDS-RAEB not in remission prior to admission for transplant - HLA 8/8 matched related or unrelated donor available - Suitable candidate for myeloablative or reduced intensity conditioning allogeneic HSCT using PBSC or marrow as stem cell resource - ECOG performance status of 0-2 - Normal organ function Exclusion Criteria: - Pregnant or breastfeeding - Leukemia with active CNS involvement - Positive HIV or HTLV-1 serology - Prior allogeneic stem cell transplantation - History of allergic reactions to compounds of similar chemical or biologic composition to GM-CSF - Uncontrolled intercurrent illness - History of different malignancy except if disease free for at least two years or cervical cancer in situ, basal or squamous cell carcinoma of the skin diagnosed and treated within previous two years
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