Phase II Study of RAD001monotherapy in Patients With Unresectable Adenoid Cystic Carcinoma
Although the histologic appearance of adenoid cystic carcinoma is low grade, management of
this malignancy is a distinct therapeutic challenge because of its insidious local growth
pattern, propensity for perineural involvement, tendency for distant metastasis, and
pronounced ability to recur over a prolonged period.
In prospectively performed clinical trials, objective responses to any cytotoxic agent or
regimen are infrequent, whereas stabilization of disease was observed more commonly.
In adenoid cystic carcinoma, the study focusing on PI3-K/AKT/mTOR pathway is rare.
According to Younes MN et al's study, adenoid cystic carcinoma cell lines have increased
pAkt activity when EGF-stimulation is added. And when treated with EGFR/VEGFR TK dual
inhibitor, the phosphorylated form of Akt decreased despite of total level of Akt is
When the investigators consider that the increased pAkt activity is one of possible
predictor to mTOR inhibitor, the mTOR inhibitor might have an activity in adenoid cystic
Although mTOR is clearly an attractive therapeutic target in tumor, no clinical study on
mTOR inhibition by RAD001 has been systematically conducted in adenoid cystic carcinoma.
In phase I study of RAD001, 2 patients with adenoid cystic carcinoma show some response to
RAD001 (unpublished data).
So the investigators design this phase II study of RAD001 in adenoid cystic carcinoma to
evaluate the efficacy of RAD001 in this orphan disease.
Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
progression free survival rate at 4 months
proportion of patients who are alive and progression-free at the time of 4 months of treatment among all patients
Yung-Jue Bang, MD, PhD
Seoul National University Hospital
Korean Food and Drug Administration:Korea, Republic of