Phase II study of dichloroacetate (DCA) in combination with chemoradiation (CRT) for unresected, locally advanced squamous cell carcinoma of the head and neck (LA-SCCHN).

e17089 Background: DCA is a metabolic inhibitor that blocks pyruvate dehydrogenase kinase (PDK), which reduces tumor lactate production and may facilitate immune clearance. In vivo SCCHN mouse model data demonstrates improved treatment response with DCA in combination with cisplatin-based CRT. We performed a randomized phase II study to evaluate the safety and efficacy of this approach in patients (pts) with LA-SCCHN. Methods: Pts with previously untreated stage III-IVB SCCHN of the hypopharynx (HP), oropharynx (OP), oral cavity (OC), and larynx (L) were included. Pts were randomized 1:1 to receive DCA 12.5mg/kg or placebo (P) PO or per G-tube twice a day during CRT. CRT consisted of cisplatin 100 mg/m2 (days 1, 22, and 43) given in with radiotherapy (70 Gy/35 fractions/7 weeks). The primary outcome was safety, defined as the group difference between grade 3 and higher adverse events (AEs) based on CTCAE version 4.0. Efficacy was evaluated by overall response rate (ORR), progression-free survival (PFS), a...