The study design should be similar to the MSCs in COVID-19 ARDS study, for example:
"This will be a randomized (1:1 ratio for 3:1 as suggested by davisite), double blind, placebo controlled trial. The study is designed to have interim analyses for stopping accrual early for efficacy. Patients will be randomized in a 1:1 allocation to intravenous infusion of MSCs (remestemcel-L) plus standard of care versus placebo plus standard of care"
The trial should be a P3 given that there is no discernible difference in disease mechanisms for pediatric vs adult patients, as suggested by the ODAC committee. This may also be the reason why the FDA appears willing to accept an adult study in place of another pediatric study. Hopefully MSB will get further clarity after the Type A meeting. One significant issue is what will be the primary endpoint. Also, what criteria will MSB use to determine when standard of care is ineffective, given they will have a small window to operate in.
Nonetheless, SI seems confident.. “There’s no point dropping it when you are 90 per cent at the end and the product clearly works,” Mr Itescu said. “I would say that I’m 90 per cent confident that this small additional study in adults will get us market approval. It didn’t happen today. But it will happen next time around ... and you’ve got to show persistence and get to the end zone,” Itescu said.
(20min delay)