Early-Stage Clinical Trials Underestimate Small Molecule, Targeted Anticancer Drug Toxicity

OncoTherapy Network

Dose-determining phase I clinical trials may not correctly determine the appropriate doses of small molecule oncology drugs to administer in larger, later stage trials according to a new study published in Clinical Cancer Research.

Phase I, dose-elevating clinical trials in a small number of patients are the standard way to determine the appropriate doses to test in later stage clinical trials. These small trials analyze pharmacokinetics, pharmacodynamics, and toxicity to determine the drug doses and schedules that are most likely to balance safety and efficacy.
The current study, conducted by researchers at The Institute of Cancer Research  and the Royal Marsden Hospital NHS Foundation Trust in the United Kingdom, suggests that phase 1 dose-ranging studies do not correctly reveal the severity of side effects of these cancer agents. The result is high incidence of varying drug toxicity experienced by patients taking part in phase III oncology clinical trials testing targeted small molecule agents.......