Eric J. Sherman, MD, Head and Neck Oncology Service, Memorial Sloan-Kettering Cancer Center, discusses the phase III clinical trial that investigated vandetanib (Caprelsa) a tyrosine kinase inhibitor that is now approved by the FDA for the treatment of symptomatic or progressive medullary thyroid cancer in patients with unresectable locally advanced or metastatic disease.

The randomized phase III study compared vandetanib to placebo. If patients receiving the placebo progressed while on the first-line treatment they were allowed to crossover to the vandetanib arm of the trial. Patients receiving vandetanib saw an increase in progression-free survival but an overall survival (OS) benefit was not demonstrated. The lack of an OS benefit can be contributed to the design and crossover within the trial.

The drug elicited a higher than expected response rate and improved the quality-of-life for patients with symptomatic medullary thyroid cancer.