Anavex Life Sciences Corp (AVXL)

[Steady_T]

A careful reading of the PR suggests that the 15 person trial is to do dose titration and determine what doses are appropriate for the large placebo controlled follow on trial. The size of which was not disclosed. They rolled that into the Phase 2 trial but to me it looks like a hybrid between a phase 1 and phase 2, only using Rett girls instead of the healthy young adults used in the AU Phase 1 trial.

I can see why the FDA might take a long look at this trial protocol.

The previous phase 1 used healthy young adults and established safety and MTD. Rett girls are pediatric subjects in many cases so there are a lot of questions raised by that factor. Rett girls already have a lot of issues that make the phase 1 results less applicable because of the lack of equivalence in subjects. That placed the FDA in a difficult position. The trial needs to determine the dosages that young Rett girls can tolerate and look for drug reactions due to the disturbed body chemistry of the Rett girls. There is no readily available equivalent population to do these studies on, so the FDA approved a small population, dose titration study using Rett diagnosed girls. That limits the risk to a small population before proceeding on to a larger trial population.

I would imagine there was a LOT of discussion within the FDA about how to do that and the safety and ethical implications involved.

I vaguely remember the number 60 being mentioned in times past. Whether that is still the case or not I do not know.