They are small-scale, early human studies designed to evaluate a drug mechanism of action, pharmacokinetics, or pharmacodynamics before traditional safety/dose-escalation trials. These studies use limited human exposure -low doses, few subjects – to guide development decisions, having no therapeutic or diagnostic intent.
They are conducted very early, often as the first-in-human studies (Phase 0 or early Phase I) Their purpose is to quickly determine if a drug behaves as expected in humans (e.g., binds to the target), allowing for rapid termination of unpromising candidates or selection of the best compounds.
- FDA-https://www.fda.gov/regulatory-information/search-fda-guidance-documents/exploratory-ind-studies#:~:text=An%20exploratory%20IND%20study%20is%20a%20clinical,products%20*%20Screening%20studies%20*%20Microdose%20studies
- NIH-ClinicalTrials.gov Glossary https://clinicaltrials.gov/study-basics/glossary
