A Randomized, Multicenter, Double-Blind, Phase II Trial of a KLH Conjugated Trivalent Ganglioside Vaccine Containing GM2, GD2 Lactone, and GD3 Lactone With the Immunological Adjuvant OPT-821 Versus OPT-821 Alone in Metastatic Sarcoma Patients Who Are Rendered Disease Free
This study is a Phase II randomized, double-blind, multi-center study of a trivalent
ganglioside vaccine plus the immunological adjuvant OPT-821 (Arm A) versus OPT-821 alone
(Arm B) for patients with metastatic sarcoma at initial presentation or with relapsed
disease who have been rendered disease-free following either surgical resection or
multi-modality therapy. The primary aim of this study is to demonstrate the efficacy of
vaccine therapy over non-specific immune therapy. Another aim of this study is to obtain
sufficient data to further the development of this specific vaccine therapy as well as guide
future study designs for therapeutic cancer vaccines in general.
To be eligible, patients must have histologically confirmed sarcoma, must be clinically free
of disease after surgery or multimodality therapy, and must be within 8 weeks of completion
of such therapy. Given the limited data regarding ganglioside expression in Ewing sarcoma,
rhabdomyosarcoma, and gastrointestinal stromal tumors, patients with these sarcoma subtypes
with the exception of pleomorphic/anaplastic rhabdomyosarcoma will be excluded. Patients
must have a history of distant metastatic disease; patients with locally recurrent disease
only will not be eligible, as these patients demonstrate a different natural history from
those with metastatic disease.
All treatment will be performed in the outpatient setting. Patients will be randomized in a
1:1 ratio to receive a total of 10 treatments of either the vaccine plus OPT-821 (Arm A) or
OPT-821 alone (Arm B). Treatment will be administered on Visit Weeks 1, 2, 3, 8, 16, 28, 40,
52, 68, and 84. All patients will receive 150 mcg of OPT-821.
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Prevention
Progressions Free Survival
The primary objective is to compare the progression-free survival (PFS) over time.
Richard Carvajal, M.D.
Memorial Sloan-Kettering Cancer Center
United States: Food and Drug Administration
|Memorial Sloan-Kettering Cancer Center||New York, New York 10021|
|Washington University School of Medicine||Saint Louis, Missouri 63110|
|Cleveland Clinic||Cleveland, Ohio 44195|
|UCLA Medical Center||Los Angeles, California 90095-7059|
|Dana Farber Cancer Institute||Boston, Massachusetts 02115|
|Oregon Health & Sciences University||Portland, Oregon 97201|
|University of Iowa Hospitals and Clinic||Iowa City, Iowa 52242|
|University of Michigan Hospital||Ann Arbor, Michigan 48109|
|University of Colorado (Denver)||Denver, Colorado 80045|
|Winship Cancer Institute at Emory Midtown||Atlanta, Georgia 30308|
|Northwestern University - Robert H. Lurie Comprehensive Cancer Center||Chicago, Illinois 60611|
|University of Pittsburg Hillman Cancer Center||Pittsburgh, Pennsylvania 15232|
|Fred Hutchinson Cancer Research Center/Seattle Cancer Care||Seattle, Washington 98109|