Everolimus Versus Placebo in Head and Neck Cancer

Phase 2 Terminated 52 enrolled University of Chicago Novartis Pharmaceuticals · collab

RandomizedParallel-groupTriple-blindTreatment

Summary

Primary: Two-year progression-free (tumor does not grow or spread) survival in subjects treated with everolimus versus placebo after definitive local therapy.

Timeline

Start: 2010-04
Primary completion: 2018-04
Completion: 2018-10

Outcome

Missed primary endpoint

Stopped (Enrollment): “Slow accrual”

paper PFS favored everolimus, but was not significant [log-rank P = 0.093; HR = 0.44; 95% confidence interval (CI), 0.17-1.17]. PMID 36194164 ↗

registry analysis (superiority test); Everolimus (RAD001) vs Placebo; p = 0.90; Difference in survival proportions 0.0022 (95% CI -0.33 to 0.33); Comparison of Kaplan-Meier estimates NCT01111058 ↗

Publications

Nathan CO, Hayes DN, Karrison T, Harismendy O, Flores JM, Moore-Medlin T, Vokes EE, Gutkind JS, Neupane P, Mills G, Sargi Z, Seiwert T, Grilley-Olson J, Day T, Gillison M, Wade JL, Feldman L, Jha G, Kozloff M, O'Leary M, Worden FP, Cohen EEW. A Randomized Multi-institutional Phase II Trial of Everolimus as Adjuvant Therapy in Patients with Locally Advanced Squamous Cell Cancer of the Head and Neck. Clin Cancer Res. 2022 Dec 1;28(23):5040-5048. doi: 10.1158/1078-0432.CCR-21-4290.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Everolimus	Small molecule	10 mg	Oral