Wednesday, December 02, 2009

Trial as of Nov2009

Jerry Maguire, chief investigator of the Pagoclone trial, was on As I speculated, most patients from the Phase IIb trial have completed the trial period. In two months' time, all will have completed. So that would take us to the end of the year. I forgot the fact of the data being locked and the blinds only being removed once all results are in. That definitely makes sense. In a double blind trial, neither the patient nor the reseacher know which pills are placebo, i.e. a sugar pill, and which contain the to-be-tested compound. So they do not know more internally than we do. However, from the outcome measurements, they can already take averages across all participants (from high dosis to no dosis placebo). You should be able to look at the time evolution. I would predict that the outcome went positive after the first testing and then slowly declined.

Jerry expects the results in late winter. That sounds reasonable. However, do not expect a serious debate at that moment, because they will mostly likely only release a statement but not a scientific paper or the raw data (unless there is a FDA requirement to do so). Jerry also announced that the Phase IIa has now been accepted for scientific publication. The publication has taken far too long for a serious debate. Who cares about these results now? :-( The Phase IIa are presumably better controlled for loopholes and a bigger sample. The publication process in medicine is really a scandal. They should start doing pre-prints like in physics.

TEVA, an Israeli company, owns the destiny to Pagoclone, and it will be up to them to decide whether to go for an expensive Phase III trial. So what happens if Phase IIb is successful? Jerry talks about there being a Phase III before the FDA accepts Pagoclone as a stuttering medication. However, I guess you can probably get it off-label. Especially because the side effect seems to be very moderate. Peter and Eric asked about clinical significance. At what point is Pagoclone successful? 30% decrease? Or more? That's a good question. 30% is a lot for severe stutterers, but for someone who just stutters occassionally and fears such situations things won't change much!

I was struck by Jerry's subtle change of takt. He was more sober than usual about the prospects of Pagoclone. My feeling is that Pagoclone is not going to be the cure that some are looking for. Might even be a mirrage. Sorry. Jerry keeps on saying that it should be combined with psychotherapy. I agree, but only if Pagoclone is shown to have some efficacy.

What I am most interested in is the placebo effect over the long-term. It is levelling off to zero. That would be good news for the reliability of such trials. If not, does placebo really reduces our stuttering long-term or is it the design and act of measuring that makes it look as if there is an improvement?

Plenty more news and a critical review coming on this blog!

1 comment:

Lorenza said...

Hi Tom,

What is your estimate on when Pagoclone will be released to the public?