Mono Centre, Open Label Proof of Concept Study SOM230 in Progressive Medullary Thyroid Cancer Patients and the Combination With RAD001 Upon Progression
Medullary thyroid cancer (MTC) is a neuroendocrine tumor originating from thyroid C cells.
Neuroendocrine tumors have been demonstrated to express somatostatin receptors as well as
mTOR pathway. The somatostatin analogues now available (octreotide and lanreotide) act
preferentially through the somatostatin receptor subtype 2 (sst2). In MTC, these compounds
have been reported to exert anti-secretive effects on calcitonin but no anti-proliferative
effects.SOM230 (pasireotide) is a new somatostatin analogue showing a peculiar binding
profile with high affinity for sst1, sst2, sst3, sst5. Preliminary data show SOM230 to be
effective in a phase II study on patients with metastatic carcinoid. RAD001 (everolimus) is
a novel agent that interacts with mTOR. It was demonstrated to inhibit tumor growth in
neuroendocrine tumor cell lines. Some clinical trials have explored the efficacy of a
combined therapy with RAD001 plus octreotide in patients with digestive neuroendocrine
tumors, highlighting encouraging results in term of tumor control.In particular, octreotide
and RAD001 seem to show a synergistic activity in inhibiting neuroendocrine tumor
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Efficacy of SOM230 in patients with progressive metastatic or postoperative persistent medullary thyroid cancer
Efficacy is defined as progression-free survival (PFS), according to RECIST criteria, in patients treated with SOM230.
From date of start therapy until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 6 months.
"Federico II" University of Naples, Italy
Italy: Ethics Committee