Phase I Clinical Study of Oral Olaparib in Pediatric Patients with Refractory Solid Tumors: Study Protocol

Overview

Journal BMC Pediatr

Publisher Biomed Central

Specialty Pediatrics

Date 2019 Jan 28

PMID 30684955

Citations 8

Authors

Masatoshi Takagi

Chitose Ogawa

Yuki Aoki-Nogami

Tomoko Iehara

Eri Ishibashi

Minoru Imai

Tetsuro Kihara

Kiyoshi Nobori

Kazuhisa Hasebe

Shuki Mizutani

Toshimi Kimura

Masashi Nagata

Masato Yasuhara

Kenichi Yoshimura

Pariko Yorozu

Hajime Hosoi

Ryuji Koike

Affiliations

Soon will be listed here.

Abstract

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 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.

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