I’m preparing for a clinical First-In-Human (FIH) study and trying to determine the optimal number of participants needed to ensure that I can get a statistically valid answer to my research question.
What factors should I consider when calculating the sample size, such as safety endpoints, dose escalation, or variability in response? Are there specific guidelines or best practices to follow when determining the right number of participants for this type of study?
Hi Rosa, I would recommend to check the EMA guidance document:
Guideline on strategies to identify and mitigate risks for first-in-human and early clinical trials with investigational medicinal products
Regards,
Joanna
Guidelines are (for a reason) not very specific on this. In traditional alternating dose escalation designs, 6-9 subjects are treated per dose. These numbers allow for sufficient placebo’s at the end, depending on the number of escalation steps. No formal statistics, but descriptive stats and graphical displays are normally used. It is one the nice aspects of phase 1: you are still allowed to look at the data, think about them and draw your own conclusions directly from the data