Apparently Santher and CHOP are excluding boys who use prednisone or deflazacort from their Idebennone trial. Anyone know why? It seems unethical.
I doubt anyone is going off steroids to participate in the study. There are plenty of boys out there not on steroids, and people have there reasons for this, ,...and I imagine it's never an easy decision.
This makes some sense, but these are children. Half of them are getting what is certain to be suboptimal care and, by defnition, they can't give informed consent. To justify making that decision for these people would require extraordinarily strong evidence. I recall that the prior study was pretty small. For example, how did they exclude the possibility that the variation in the increase in pulmonary function was the result of some confounding variable like mutations that were more or less associated with cardiac dysfunction?