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Vaccine Trial for Clear Cell Sarcoma, Pediatric Renal Cell Carcinoma, Alveolar Soft Part Sarcoma and Children With Stage IV Melanoma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00258687
Recruitment Status : Active, not recruiting
First Posted : November 28, 2005
Last Update Posted : July 7, 2020
Boston Children's Hospital
Information provided by (Responsible Party):
F. Stephen Hodi, MD, Dana-Farber Cancer Institute

Brief Summary:
The purpose of this study is to learn if a vaccine made from the patient's own tumor cells, then genetically modified to secrete granulocyte-macrophage colony-stimulating factor (GM-CSF), will delay or stop the growth of the tumor. It will also look at the vaccine's effects on the immune system and the side effects of giving a vaccine made from a subject's own cancer cells.

Condition or disease Intervention/treatment Phase
Sarcoma, Clear Cell Sarcoma, Alveolar Soft Part Renal Cell Carcinoma Melanoma Biological: GVAX Phase 1

Detailed Description:

The patient will have surgery to remove a portion of the tumor. This tumor is then brought to a special, certified laboratory where it is broken up into single cells and then washed.

Specially trained laboratory technicians then use a method known as adenoviral mediated gene transfer, which adds a new gene to the cancer calls. This gene causes the cells to make GM-CSF, a powerful hormone that stimulates the immune system. The cells are then given enough radiation so that they will never grow, but not enough to completely destroy them, developing a vaccine.

The patient is then injected with the vaccine on days 0, 7, 14, 28, and then every two weeks until the supply of vaccine has run out. The amount of vaccine that can be made depends upon the total amount of cells taken from the tumor. The actual injections are like childhood vaccinations that go under the skin or into muscle and a different place will be used for each injection.

It is hoped that the cancer cells that have been made to secrete the hormone GM-CSF will cause the patient's immune system to attack the cancer in other parts of the body.

If the tumor yields enough cells, the patient will also be given an injection of non-transduced irradiated tumor cells. Non-transduced means that the gene for GM-CSF has not been added to these cells as it has for the vaccines. This is done to measure the amount of reaction of the immune system caused by the vaccine. This injection is measuring delayed type hypersensitivity, or DTH.

The patient will be asked to undergo optional skin biopsies of the vaccine and DTH sites to see if an immune reaction is occuring at the injection sites 2 days after vaccine 1 and vaccine 5.

The following tests and procedures will be performed through out the study: physical exam, blood samples, immune studies, vital signs and physical exam.

At week 10 in the patient's treatment, or earlier if the doctor feels it is necessary, the patient will undergo a chest, abdomen and pelvic XT scan. A brain MRI will be performed if there were any abnormalities on the first brain MRI or if any new central nervous system symptoms have developed.

If the patient's disease has not disappeared or if new lesions have been found after the patient receives at least six vaccines, they may have the opportunity to undergo a second course of study treatment.

Patients may participate in this study until one of the following happens: All vaccine created from the tumor has been given to the patient; the patient's disease worsens; the patient experiences an unacceptable and/or harmful side effect; the patient becomes pregnant; the patient is unable to follow the study plan; or the patient's doctor feels it is no longer in the best interest of the patient to continue.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 12 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I Trial of Vaccination With Autologous, Lethally Irradiated Tumor Cells Engineered by Adenoviral Mediated Gene Transfer to Secrete Granulocyte-Macrophage Colony Stimulating Factor in Pediatric and Adult Patients
Actual Study Start Date : January 2005
Actual Primary Completion Date : July 2006
Estimated Study Completion Date : December 2020

Arm Intervention/treatment
Experimental: Treatment Arm A
GVAX for Sarcoma / Renal Cell Patients
Biological: GVAX
4 vaccines every two weeks

Experimental: Treatment Arm B
GVAX for Pediatric Melanoma Patients
Biological: GVAX
4 vaccines every two weeks

Primary Outcome Measures :
  1. To determine the safety and feasibility of preparation and administration of vaccine in patients with metastatic or locally advanced clear cell sarcoma (CCS), alveolar soft part sarcoma (ASPS) and translocation associated renal cell carcinoma (RCC) [ Time Frame: Years ]

Secondary Outcome Measures :
  1. To determine the disease response, immune response, and overall survival rate

Information from the National Library of Medicine

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, Learn About Clinical Studies.

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • ECOG performance status 0 or 1
  • Estimated life expectancy of greater than 6 months
  • Greater than or equal to 4 weeks from chemotherapy, radiotherapy, immunotherapy, or systemic glucocorticoid therapy
  • Greater than or equal to 6 months from prior bone marrow or peripheral blood stem cell (PBSC) transplant
  • Histologically confirmed alveolar soft part sarcoma or clear cell sarcoma at any age.
  • Evidence of metastatic disease, including having spread either to distant sites that may include brain metastases, or to regional lymph nodes alone, or locally advanced primary lesion that is not fully surgically resectable at study entry.
  • Histologically confirmed Stage IV renal cell carcinoma (patients with brain metastases still eligible)
  • Any patients with Stage IV renal cell carcinoma under the age of 25 years who do not have a renal cell carcinoma predisposition syndrome
  • Patients with Stage IV melanoma and under the age of 18 years

Exclusion Criteria:

  • Uncontrolled active infection
  • Pregnancy or nursing mothers
  • Infection with HIV, hepatitis B or hepatitis C
  • Any other significant medical, surgical, or psychiatric condition that may interfere with compliance with protocol regimen
  • Other current malignancies apart from any in situ cancer or basal or squamous cell carcinoma
  • Pediatric melanoma only: infants with transplacentally acquired melanoma; or children with brain metastases and malignant melanoma.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00258687

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United States, Massachusetts
Children's Hospital Boston
Boston, Massachusetts, United States, 02115
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02115
Sponsors and Collaborators
Dana-Farber Cancer Institute
Boston Children's Hospital
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Principal Investigator: F. Stephen Hodi, MD Dana-Farber Cancer Institute
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Responsible Party: F. Stephen Hodi, MD, Principal Investigator, Dana-Farber Cancer Institute Identifier: NCT00258687    
Other Study ID Numbers: 05-115
First Posted: November 28, 2005    Key Record Dates
Last Update Posted: July 7, 2020
Last Verified: July 2020
Keywords provided by F. Stephen Hodi, MD, Dana-Farber Cancer Institute:
Adenoviral mediated gene transfer
Pediatric Melanoma
Additional relevant MeSH terms:
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Carcinoma, Renal Cell
Sarcoma, Alveolar Soft Part
Sarcoma, Clear Cell
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Neuroendocrine Tumors
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms, Nerve Tissue
Nevi and Melanomas
Neoplasms, Connective and Soft Tissue
Kidney Neoplasms
Urologic Neoplasms
Urogenital Neoplasms
Neoplasms by Site
Kidney Diseases
Urologic Diseases
Neoplasms, Muscle Tissue
Neoplasms, Connective Tissue