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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

Phase 1
Open (Enrolling)
Sarcoma, Clear Cell, Sarcoma, Alveolar Soft Part, Renal Cell Carcinoma, Melanoma

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Trial Information

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

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.

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

- Pediatric melanoma only: infants with transplacentally acquired melanoma; or children
with brain metastases and malignant melanoma.

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

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)

Outcome Time Frame:


Safety Issue:


Principal Investigator

F. Stephen Hodi, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Dana-Farber Cancer Institute


United States: Food and Drug Administration

Study ID:




Start Date:

January 2005

Completion Date:

September 2013

Related Keywords:

  • Sarcoma, Clear Cell
  • Sarcoma, Alveolar Soft Part
  • Renal Cell Carcinoma
  • Melanoma
  • GVAX
  • GM-CSF
  • Adenoviral mediated gene transfer
  • Pediatric Melanoma
  • Carcinoma
  • Adenocarcinoma, Bronchiolo-Alveolar
  • Carcinoma, Renal Cell
  • Melanoma
  • Sarcoma, Clear Cell
  • Sarcoma, Alveolar Soft Part
  • Sarcoma



Dana-Farber Cancer Institute Boston, Massachusetts  02115
Children's Hospital Boston Boston, Massachusetts  02115