Early phase clinical trials of anticancer agents in children and adolescents - an ITCC perspective.
Date
2017-08ICR Author
Author
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
Innovative Therapies for Children with Cancer (ITCC) Consortium
Type
Journal Article
Metadata
Show full item recordAbstract
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.
Collections
Subject
Innovative Therapies for Children with Cancer (ITCC) Consortium
Humans
Neoplasms
Antineoplastic Agents
Adolescent
Child
Adolescent Health Services
Child Health Services
Societies, Medical
Europe
Clinical Trials as Topic
Research team
Paediatric Drug Development and Clinical Trials
Language
eng
License start date
2017-08
Citation
Nature reviews. Clinical oncology, 2017, 14 (8), pp. 497 - 507