A Phase II Study of Oral Cyclophosphamide and Sirolimus (OCR) in Advanced Sarcoma
The purpose of this Phase II study you are being asked to participate in will assess the
effectiveness of the combination of oral cyclophosphamide and sirolimus in sarcoma patients
with relapsed or widespread disease who cannot be cured by surgery, radiation or
conventional chemotherapy. Malignant connective tissue tumors of soft tissue and bone
(sarcomas) are highly aggressive cancers. There are few available chemotherapy treatments
that are active in treating sarcomas. Sarcomas that have metastasized (spread throughout the
body) are usually fatal. There is a great need to identify new active drugs to treat
metastatic or relapsed sarcomas. Low dose oral daily cyclophosphamide is an established
chemotherapy regimen for treatment of malignant and autoimmune disease and is generally well
tolerated. Sirolimus is approved for prevention of kidney rejection after transplantation.
Temsirolimus, a form of sirolimus, is approved for the treatment of kidney cancer.
Sirolimus combined with cyclophosphamide in animal models of sarcoma resulted in significant
anti-tumor activity. Tumor and blood samples will be studied to look for known protein
targets of the medication to help learn why certain subjects have a favorable response to
Allocation: Non-Randomized, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Patients who were evaluable for response to therapy, alive and without evidence of sarcoma disease progression. Target lesions followed were lesions that had progressed by World Health Organization (WHO) criteria.
Scott Schuetze, MD, PhD
University of Michigan Cancer Center
United States: Food and Drug Administration
|University of Michigan||Ann Arbor, Michigan 48109-0624|