Background: Phase 1 clinical trials based on pre-clinical data and animal toxicology are associated with the highest uncertainty. They remain, however, a critical first step in a drug development. In oncology phase 1 studies are carried out usually on patients who have previously received unsuccessful systemic therapy and for whom no standard therapy exists. The average age of patients participating in phase 1 trial is about 60 years.
Despite the fact that cancer is mostly a disease of adults, it remains one of the major causes of death in pediatric populations. As cancer in children differs from cancer in adults (i.e. children's tumors differ histologically from adults’) data based on pediatric trials are needed. Phase 1 pediatric trials in oncology almost always follow after initial testing in adults, and use a starting dose defined in relation to adults' maximum tolerated dose (MTD). Since they involve populations that are unable to provide valid informed consent, phase 1 pediatric trials in oncology are ethically contentious. Furthermore, there is uncertainty as to whether the risk/benefit balance in phase 1 studies should count as therapeutic.
Objectives: Quantify the average risk/benefit balance for pediatric phase 1 cancer studies, measuring benefit by response rate and risk by Grade 3,4, or 5 drug related events.
Methods: PubMed and EMBASE were systematically searched in order to identify full-length phase I pediatric trials in oncology published between 2004-2015.
Results: 6579 publications were identified. We are going to screen titles and abstracts in order to include those that meet the inclusion criteria. The remaining trials will provide the basis for our detailed analysis. We expect to finish this analysis by June 2015.
Conclusions: To be determined by June 2015.