This is part 3 of a 3-part series on recent pressing topics affecting Phase I trials. Click here to read Part 1: "How Good is the Data on ClinicalTrials.gov". Click here to read Part 2: "Why a Recent FDA Brief on Clinical Cancer Research Is Really About Much More Than Cancer Research".
I have recently put a couple of blogs out on the size of Phase 1 clinical trials - the first was digging into the data quality of CT.g and the second on the FDA acceptance of expanded cohort studies, or Phase 1E as we call them. We commented that the FDA acknowledges having “3 dozen” such studies. It piqued our interest so we dug into the data ourselves, using publicly available data on ClinicalTrials.gov.
In short, there are not 3 dozen of these studies but exactly 96. When we dug in further we found a large number of registries and observational studies mislabeled as Phase 1. When these are filtered out we are left with 58 Phase I interventional studies. The largest study is recorded as having 99,999,999 run by the National Heart, Lung, and Blood Institute (NHLBI) NCT00001529. This is farcical since this study will not enroll this number of subjects. The organizers, who are part of the NIH should provide more informed estimates, if nothing else than for IRB and ethical issues. The largest Pharmaceutically sponsored Phase I study is by Astra Zeneca, entitled “Autoinjector Device Assessment Study” of 3052 subjects, but the current prize for largest Phase 1E study goes to EMD Serono (now Serono) with an anticipated enrollment of 1670 subjects in a study entitled “Avelumab in Metastatic or Locally Advanced Solid Tumors (JAVELIN Solid Tumor).”
Look through the largest Phase I trials for yourself - we've compiled information about the 101 studies with 800 or more subjects into an excel workbook, which is available for download by submitting this form.