Multi-Institutional Randomized Double-Blind Phase II Trial of Everolimus Vs. Placebo As Adjuvant Therapy in Patients with Locally Advanced Squamous Cell Cancer of the Head and Neck (SCCHN)

The dismal 5-year survival rate for advanced stage smoking related SCCHN of <30% has not changed in the past 30 years. Akt/mTOR is activated in most SCCHN and pathway activation in surrounding normal mucosa is associated with recurrences. Oral mTOR inhibitors appear well tolerated and effective in window of opportunity SCCHN trials. The purpose of this trial (NCT01111058) was to determine whether adjuvant everolimus improves 2-year progression-free survival (PFS) in patients with advanced SCCHN and investigate correlative biological factors associated with response.