Phase-0 Including Microdosing Approaches

Phase-0 drug development trials are clinical development approaches that include microdosing trials. These approaches are sometimes called ‘Exploratory Clinical Trials’ ‘Exploratory Investigational New Drug (eIND)’ applications. Phase-0 studies are aimed at providing human data to support selection of preclinical drug candidates prior to entering clinical development. Phase-0 trials use limited exposure to the drug (lower dose and/or shorter duration of exposure) than traditional Phase-1. Because of their inherent safety Phase-0 studies require less preclinical data than traditional Phase-1 studies. Thus, they enable obtaining first-in-human data in a safer, quicker, and cheaper manner. The main benefit of Phase-0 studies is the potential termination of a non-viable drug sooner than would otherwise be possible. Check our recent publication in Nature Reviews Drug Discovery at:

Phase-0 approaches require sensitive analytical tools such as Accelerator Mass Spectrometer (AMS), Positron Emission Tomography (PET), and Liquid Chromatography Tandem Mass Spectrometry (LC-MS/MS). These tools enable the detection of the low concentrations of the drug in body organs and tissues. Phase-0 trials have many similarities with other clinical trials. These include the need for ethical and regulatory approval, the use of human research participants (healthy volunteers or patients) and the need for a detailed clinical study design. Advantages of Phase-0 include the ability to enter directly into testing in patients, the ability to administer all drugs intravenously (IV), and the ability to use cassette microdosing. In cassette microdosing multiple drug candidates are given simultaneously to the same research participant.

The Phase-0/Microdosing Network is a non-profit organization dedicated to the education, research, development of Phase-0 including microdosing approaches and to clarifying their potential role in drug development decision-making. The membership of the Phase-0/Microdosing Network includes individuals from a range of disciplines that are critical to the effectiveness of Phase-0 including microdosing trials in drug development. These include experts in clinical pharmacology, clinical research, regulatory guidelines, statistics, modeling and simulations, in-vivo and in-vitro pre-clinical studies, economics of drug development, analytical tools, medicinal chemistry, radiochemistry, and biomarkers. Members come from industry, academia, regulatory agencies, drug development sponsors, clinical research contract research organizations (CROs), non-profit and patient advocacy organizations.

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