Phase II clinical trial of sorafenib in metastatic medullary thyroid cancer.

ABSTRACT:

Purpose

Mutations in the RET proto-oncogene and vascular endothelial growth factor receptor (VEGFR) activity are critical in the pathogenesis of medullary thyroid cancer (MTC). Sorafenib, a multikinase inhibitor targeting Ret and VEGFR, showed antitumor activity in preclinical studies of MTC.

Patients and methods

In this phase II trial of sorafenib in patients with advanced MTC, the primary end point was objective response. Secondary end points included toxicity assessment and response correlation with tumor markers, functional imaging, and RET mutations. Using a two-stage design, 16 or 25 patients were to be enrolled onto arms A (hereditary) and B (sporadic). Patients received sorafenib 400 mg orally twice daily.

Results

Of 16 patients treated in arm B, one achieved

SUBMITTER: Lam ET 

PROVIDER: S-EPMC2881718 | biostudies-literature | 2010 May

REPOSITORIES: biostudies-literature