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.
Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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.
Kenneth G. Lucas, MD
Milton S. Hershey Medical Center
United States: Food and Drug Administration