To determine the response rate (RR) of metastatic or locally advanced
pheochromocytoma/paraganglioma to axitinib administered daily.
Determine the progression-free survival.
In an exploratory manner examine the extent of activation of the VEGFR pathway in
pheochromocytoma/paraganglioma using a semi-quantitative immunohistochemistry assay and
examine the relationship with response to therapy.
Perform pharmacogenomics analyses of drug metabolism and transport proteins through
germline DNA examination.
The long-term survival for malignant pheochromocytoma/paraganglioma (PHEO/PGL) may be limited
because of its hormonal effects as well as its aggressive behavior and dissemination,
particularly in some hereditary PHEO/PGL. Although several therapeutic modalities have been
used to palliate malignant PHEO/PGL, a continued search for new agents to address the
malignancy is needed to improve outcomes. One approach is to utilize drugs that target
signaling pathways leading to decreased proliferation and survival of cancer cells. Some data
in the literature suggests that in malignant PHEO/PGL, VEGF seems to play a role in the
biology of the cancer and thus, its inhibition could reduce tumor growth. Sunitinib and
imatinib has been used in a limited number of malignant pheochromocytomas with varying
responses. Axitinib was designed to inhibit VEGFR that participates in tumor angiogenesis. In
order to determine the activity of axitinib in tumor and hormonal responses in malignant
PHEO/PGL, it will be used as a single agent in this study. The combination of
cyclophosphamide; vincristine and dacarbazine have been shown to produce partial responses in
malignant PHEO/PGL. The majority of the patients may have already received this combination
or will receive this combination chemotherapy in the future. The goal is to develop multiple
lines of effective treatments for this disease.
If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
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