Designing a seamless phase II/III clinical trial using early outcomes for treatment selection: An application in multiple sclerosis.

A new trial design for doing neuroprotective MS clinical studies. The investigators propose using an adaptive design in that the trial changes over time so that only the winners (2 out of 4 of the active arms) in the first phase get through the final or definitive arm of the study.

“This study design is very appealing, but unfortunately it may go down poorly in the patient community. The study will take about 7 years to complete; 2 years to recruit, 2 years to do phase 1 and 3 years for the phase 2 arm of the study. In my opinion 7 years is simply too long in the life of someone with progressive MS to get an answer. We clearly need better and quicker trials for testing neuroprotective drugs in progressive MS.”

Friede et al. Stat Med. 2011 Feb 22. doi: 10.1002/sim.4202. [Epub ahead of print]

COI: I sit on the steering committee of the UK MS Society’s Clinical Trial Network that commissioned the work to design this study.

Leave a Reply

%d bloggers like this: