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BMC Pediatrics
Published in: BMC Pediatrics 1/2019

Open Access 01-12-2019 | Ewing's Sarcoma | Study protocol

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

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

Published in: BMC Pediatrics | Issue 1/2019

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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/m2 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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Metadata
Title
Phase I clinical study of oral olaparib in pediatric patients with refractory solid tumors: study protocol
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
Publication date
01-12-2019
Publisher
BioMed Central
Published in
BMC Pediatrics / Issue 1/2019
Electronic ISSN: 1471-2431
DOI
https://doi.org/10.1186/s12887-019-1409-7

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