Journal Article (Review Article)

DKFZ-2017-01521

http://join2-wiki.gsi.de/foswiki/pub/Main/Artwork/join2_logo100x88.png Early phase clinical trials of anticancer agents in children and adolescents - an ITCC perspective.

Moreno, L. ; Pearson, A. D. J. ; Paoletti, X. ; Jimenez, I. ; Geoerger, B. ; Kearns, P. R. ; Zwaan, C. M. ; Doz, F. ; Baruchel, A. ; Vormoor, J. ; Casanova, M. ; Pfister, S.DKFZ* ; Morland, B. ; Vassal, G. ; Cancer, I. T. f. C. w. (Collaboration Author)

2017
Nature Publ. Group New York, NY

This record in other databases:  

Please use a persistent id in citations: doi:10.1038/nrclinonc.2017.59

Abstract: 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.

Keyword(s): Antineoplastic Agents

---

Contributing Institute(s):

1. B062 Pädiatrische Neuroonkologie (B062)
2. DKTK Heidelberg (L101)

Research Program(s):

1. 312 - Functional and structural genomics (POF3-312) (POF3-312)

Appears in the scientific report 2017

---

Database coverage:
; Current Contents - Clinical Medicine ; IF >= 15 ; JCR ; Nationallizenz ; SCOPUS ; Science Citation Index ; Science Citation Index Expanded ; Thomson Reuters Master Journal List ; Web of Science Core Collection

---