Sample Size
death, coronavirus disease 2019
CORONAVIRUS DISEASE 2019, EVENT, RECRUITMENT, SECONDARY, EVENTS
No formal statistical power calculation was performed. Even as single adjunctive therapies, there were limited available data on the use of these drugs in TBM to predict the likely rate of AESI and/or death. Given this would be further complicated when considering likely event rate when combined, it was felt that it would be more pragmatic to create a recruitment target of 100 participants with frequent blinded review of cumulative safety events by an independent DSMB. A secondary aim of LASER-TBM was to serve as a planning study to generate PK and safety data to inform a phase 3 RCT of intensified treatment in TBM (NCT04145258), which in part would influence resulting sample size.The decision to stop recruitment prior to 100 participants enrolled was due to the rate of recruitment being slower than anticipated due to the coronavirus disease 2019 pandemic; funding for the trial was due to cease in March 2021 and therefore recruitment could take place up until January 2021; and DSMB review of safety data (both during recruitment to the trial and following enrollment of last recruit) had revealed no reason for the planned RCT to not go ahead.