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GVAX vs. Placebo for MDS/AML After Allo HSCT

This study is currently recruiting participants.
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Verified June 2016 by Vincent T. Ho, MD, Dana-Farber Cancer Institute
Information provided by (Responsible Party):
Vincent T. Ho, MD, Dana-Farber Cancer Institute Identifier:
First received: January 8, 2013
Last updated: September 19, 2016
Last verified: June 2016

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 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) 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 form 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 transplant.

Condition Intervention Phase
Myelodysplastic Syndrome Acute Myeloid Leukemia Biological: GVAX Biological: Placebo Vaccine Procedure: Allogeneic Hematopoietic Stem Cell Transplant Drug: Busulfan Drug: Fludarabine Drug: Tacrolimus Drug: Methotrexate Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Randomized Placebo-controlled Phase II Trial of Irradiated, Adenovirus Vector Transferred GM-CSF Secreting Autologous Leukemia Cell Vaccination (GVAX) Versus Placebo Vaccination in Patients With Advanced MDS/AML After Allogeneic Hematopoietic Stem Cell Transplantation

Resource links provided by NLM:

Further study details as provided by Vincent T. Ho, MD, Dana-Farber Cancer Institute:

Primary Outcome Measures:
  • Progression Free Survival [ Time Frame: 18 months ]
    Progression-Free Survival (PFS) at 18 months after randomization

Secondary Outcome Measures:
  • Safety and toxicity of vaccination, as measured by the development of grade II-IV and III-IV acute graft versus host disease, CTC grade 3 and higher non-hematologic toxicity, or grade 4 and higher hematologic toxicity attributable to vaccination [ Time Frame: 2 years ]
    Assessment of the safety of vaccination following allogeneic (ablative or reduced intensity) stem cell transplantation

  • Incidence of acute and chronic GVHD [ Time Frame: 2 years ]
    Assessment of incidence fof grade 2-4 and grade 3-4 acute GVHD, and chronic GVHD after vaccination following allogeneic stem cell transplantation

  • PFS after start of vaccination [ Time Frame: 2 years ]
    Assessment of PFS after start of vaccination

  • Relapse and non-relapse mortality [ Time Frame: 2 years ]
    Assessment of relapse and non-relapse mortality after vaccination

  • Overall Survival [ Time Frame: 2 years ]
    Assessment of overall survival after vaccination

  • Biologic activity of GVAX vs. Placebo as assessed by laboratory and histologic analyses of blood before and after vaccination. [ Time Frame: 2 years ]
    Assessment of the biologic activity of GVAX as compared to placebo vaccination after HSCT

Estimated Enrollment: 152
Study Start Date: January 2013
Estimated Study Completion Date: January 2033
Estimated Primary Completion Date: July 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: GVAX
GVAX vaccine
Biological: GVAX Procedure: Allogeneic Hematopoietic Stem Cell Transplant Drug: Busulfan Drug: Fludarabine Drug: Tacrolimus Drug: Methotrexate
Placebo Comparator: Placebo
Placebo vaccine
Biological: Placebo Vaccine Procedure: Allogeneic Hematopoietic Stem Cell Transplant Drug: Busulfan Drug: Fludarabine Drug: Tacrolimus Drug: Methotrexate

  Show Detailed Description


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • AML, MDS-RAEB not in remission prior to admission for transplant
  • HLA 8/8 or 7/8 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
  • 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 the past two years
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01773395

Contact: Vincent Ho, MD 6176325938

United States, Massachusetts
Brigham and Women's Hospital Recruiting
Boston, Massachusetts, United States, 02115
Contact: Vincent Ho, MD    617-632-5938   
Principal Investigator: Vincent Ho, MD         
Dana-Farber Cancer Institute Recruiting
Boston, Massachusetts, United States, 02115
Contact: Vincent Ho, MD    617-632-5938   
Principal Investigator: Vincent Ho, MD         
Beth Israel Deaconess Medical Center Not yet recruiting
Boston, Massachusetts, United States, 02215
Contact: David Avigan, MD    617-667-9920   
Principal Investigator: David Avigan, MD         
Sponsors and Collaborators
Dana-Farber Cancer Institute
Principal Investigator: Vincent Ho, MD Dana-Farber Cancer Institute
  More Information

Responsible Party: Vincent T. Ho, MD, Principal Investigator, Dana-Farber Cancer Institute Identifier: NCT01773395     History of Changes
Other Study ID Numbers: 12-217
Study First Received: January 8, 2013
Last Updated: September 19, 2016

Keywords provided by Vincent T. Ho, MD, Dana-Farber Cancer Institute:

Additional relevant MeSH terms:
Leukemia, Myeloid, Acute
Myelodysplastic Syndromes
Neoplasms by Histologic Type
Leukemia, Myeloid
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Immunologic Factors
Physiological Effects of Drugs
Abortifacient Agents, Nonsteroidal
Abortifacient Agents
Reproductive Control Agents
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Dermatologic Agents
Enzyme Inhibitors
Folic Acid Antagonists
Immunosuppressive Agents
Antirheumatic Agents
Nucleic Acid Synthesis Inhibitors processed this record on June 23, 2017