Toggle Main Menu Toggle Search

Open Access padlockePrints

Early phase clinical trials of anticancer agents in children and adolescents - an ITCC perspective

Lookup NU author(s): Professor Andrew Pearson, Dr Pamela Kearns, Professor Hermann Josef Vormoor


Full text for this publication is not currently held within this repository. Alternative links are provided below where available.


© 2017 Macmillan Publishers Limited, part of Springer Nature. In the past decade, the landscape of drug development in oncology has evolved dramatically; however, this paradigm shift remains to be adopted in early phase clinical trial designs for studies of molecularly targeted agents and immunotherapeutic agents in paediatric malignancies. In drug development, prioritization of drugs on the basis of knowledge of tumour biology, molecular 'drivers' of disease and a drug's mechanism of action, and therapeutic unmet needs are key elements; these aspects are relevant to early phase paediatric trials, in which molecular profiling is strongly encouraged. Herein, we describe the strategy of the Innovative Therapies for Children with Cancer (ITCC) Consortium, which advocates for the adoption of trial designs that enable uninterrupted patient recruitment, the extrapolation from studies in adults when possible, and the inclusion of expansion cohorts. If a drug has neither serious dose-related toxicities nor a narrow therapeutic index, then studies should generally be started at the adult recommended phase II dose corrected for body surface area, and act as dose-confirmation studies. The use of adaptive trial designs will enable drugs with promising activity to progress rapidly to randomized studies and, therefore, will substantially accelerate drug development for children and adolescents with cancer.

Publication metadata

Author(s): Moreno L, Pearson ADJ, Paoletti X, Jimenez I, Geoerger B, Kearns PR, Zwaan CM, Doz F, Baruchel A, Vormoor J, Casanova M, Pfister SM, Morland B, Vassal G

Publication type: Review

Publication status: Published

Journal: Nature Reviews Clinical Oncology

Year: 2017

Volume: 14

Issue: 8

Pages: 497-507

Online publication date: 16/05/2017

Acceptance date: 02/04/2016

ISSN (print): 1759-4774

ISSN (electronic): 1759-4782

Publisher: Nature Publishing Group


DOI: 10.1038/nrclinonc.2017.59

PubMed id: 28508875