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A Phase 1 Study to Determine the Immunologic Effects of a MAGE- A1, MAGE- A3, NY-ESO-1 Vaccine in Patients With High Risk Neuroblastoma, Osteogenic Sarcoma, and Rhabdomyosarcoma

Phase 1
1 Year
70 Years
Not Enrolling
Neuroblastoma, Rhabdomyosarcoma, Osteogenic Sarcoma

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Trial Information

A Phase 1 Study to Determine the Immunologic Effects of a MAGE- A1, MAGE- A3, NY-ESO-1 Vaccine in Patients With High Risk Neuroblastoma, Osteogenic Sarcoma, and Rhabdomyosarcoma

MAGE -A1, MAGE- A3, and NY-ESO-1 are antigens that can be found with significant frequency
on neuroblastoma, rhabdomyosarcoma, and osteogenic sarcoma, three relatively common solid
tumors that in some cases can be associated with a high risk for relapse. In this study
each subject will be screened for the presence of these antigens, and an individualized
vaccine will be developed and administered using the subject's own dendritic cells (DC).

This study consists of two phases: a screening phase and a treatment/vaccine phase. First,
eligible individuals will be consented into the screening phase. Tumor specimens will be
tested by immunohistochemistry or RT-PCR for the presence of MAGE- A1, MAGE- A3, and
NY-ESO-1. Those testing positive for one or more antigen can be consented for the treatment
phase of the study. Blood will be drawn for DC culture, and approximately one month later a
series of three vaccines will be administered at two week intervals. Subjects will receive a
topical medication called imiquimod to the vaccine site prior to and following each
injection, to help immune cells travel into the area. Study participation occurs over 18
months and also involves periodic physical examinations and blood draws.

Inclusion Criteria

Inclusion Criteria for Screening Phase:

Patient 1 to 70 years of age with neuroblastoma, rhabdomyosarcoma, or osteogenic sarcoma,
who have one or more of the following high risk features:

- Neuroblastoma:

- Stage IV disease

- Stage III disease with n-myc amplification

- Osteogenic sarcoma:

- Presence of metastases

- Elevated alkaline phosphatase or LDH at diagnosis

- Primary tumor affecting the axial skeleton

- Poor histopathological response after completion of pre-surgical
chemotherapy (≥10% viable tumor)

- Rhabdomyosarcoma:

- Stage IV disease

- Alveolar histology

- Positive tumor margins, with lymph node positivity

Inclusion Criteria for Vaccine Phase:

- Patient meets all screening criteria and tumor is positive for NY-ESO-1, MAGE- A1, or
MAGE-A3 by immunohistochemistry or RT-PCR.

- Patients who are between 3 months and 2 years following the completion of therapy,
and have achieved at least a very good partial response to primary therapy.

- No chemotherapy is planned for one month following the last vaccination.

- Bilirubin <2 mg/dL, and SGOT/SGPT <2.5 x normal

- Creatinine clearance > 50ml/min as estimated by patient's serum creatinine, weight,
and age

- Room air pulse oximetry >94%

- Patient is not pregnant

- Male and female sexually active patients of reproductive who wish to participate must
agree to use acceptable contraception

- Patient is not moribund and has a projected life expectancy >6 months

- Lansky performance scale > 70, ECOG < 2 (Appendix I)

- Potential subjects will be tested for HIV 1 and 2 antibodies, HTLV 1/2 antibodies,
and for HIV 1, hepatitis C and hepatitis B virus by NAT testing. - -Subjects testing
positive for any of these pathogens will be ineligible for vaccine.

- White blood cells ≥ 2.5 K/µL, Hemoglobin ≥ 8 g/dL, Hematocrit > 25%, and Platelets ≥
70 K/µL

- Patient does not have central nervous system involvement.

- Patient does not a have a history of autoimmune disease, specifically inflammatory
bowel disease, systemic lupus erythematosis, or rheumatoid arthritis

- Patient is not receiving concurrent systemic steroid therapy

- Patient does not have a known systemic hypersensitivity to imiquimod or any vaccine

Type of Study:


Study Design:

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

Outcome Measure:

The primary objective is to determine if there is an amplification or new development of NY-ESO-1, MAGE-A1, or MAGE-A3 specific CD4+ or CD8+ T cells post-vaccination.

Outcome Time Frame:

2 years

Safety Issue:


Principal Investigator

Kenneth G. Lucas, MD

Investigator Role:

Study Chair

Investigator Affiliation:

Milton S. Hershey Medical Center


United States: Food and Drug Administration

Study ID:

IRB 30761



Start Date:

June 2009

Completion Date:

August 2010

Related Keywords:

  • Neuroblastoma
  • Rhabdomyosarcoma
  • Osteogenic Sarcoma
  • MAGE-A1
  • MAGE-A3
  • NY-ESO-1
  • antigen
  • vaccine
  • dendritic cells
  • immunohistochemistry
  • immunology
  • Neuroblastoma
  • Osteosarcoma
  • Rhabdomyosarcoma
  • Sarcoma