Phase I clinical study of oral olaparib in pediatric patients with refractory solid tumors: study protocol

BACKGROUND: There is no established standard chemotherapy for recurrent pediatric solid tumors such as neuroblastoma and sarcoma. Since some of these tumor cells show dysfunctions in homologous recombination repair, the goal is to conduct a phase I study of olaparib, a poly(ADP-ribose) polymerase inhibitor. In this clinical trial, the aims are to evaluate the safety, tolerability, and efficacy of olaparib in pediatric patients with refractory solid tumors and to recommend a dose for phase II trials. METHODS: In this open-label, multicenter study, olaparib tablets (62.5, 125, and 187.5 mg/m(2) b.i.d.) will be administered orally in a standard 3 + 3 dose escalation design. Patients aged 3 to 18 years with recurrent pediatric solid tumors are eligible. Pharmacokinetic and pharmacodynamic analyses will also be performed. DISCUSSION: This study aims to extend the indications for olaparib by assessing its safety and efficacy in pediatric refractory solid tumor patients. TRIAL REGISTRATION: UMIN-CTR (UMIN000025521); Registered on January 4, 2017.