Phase 0 Microdosing - At the Discovery and Development Interface
Our clients typically ask us to conduct Phase 0/Human Microdosing studies for the following:
- to select the most promising compound from drug candidates for full development
- to determine whether a compound reaches its site of action, where traditional techniques such as in silico modeling or allometric scaling may be unreliable
"Because exploratory IND studies present fewer potential risks than do traditional phase I studies that look for dose-limiting toxicities, such limited exploratory IND investigations in humans can be initiated with less preclinical support than is required for traditional IND studies."
Guidance for Industry, Investigators, and Reviewers. Exploratory IND Studies U.S. Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research (CDER) January 2006.
A Phase 0 (or Human Microdose) study is performed at a very early stage of drug development in order to obtain preliminary pharmacokinetic data on a drug candidate prior to commencement of the Phase I clinical trials. As its name implies, the dose administered during a human Phase 0 study is very small, the amount being defined by both the EMEA and FDA as 100th of the predicted pharmacologic dose to a maximum of 100 μg.
These small doses are considered inherently safer than pharmacologically active doses and therefore regulatory authorities accept a much reduced safety toxicology package to allow a human Phase 0 study to proceed. This allows drug candidates to be administered to human volunteers earlier and with less expenditure compared to a Phase I clinical study.
Please follow the links below to access publications regarding Phase 0 Human Microdosing:
