RE: AZD-103/ELND-005......bioboffin
bioboffin........."IT IS NOT A BLINDED TRIAL, so presumably when they see efficacy, they will declare victory and go back to the FDA to approve another trial."
Actually bioboffin it IS a Phasell Blinded Trial..... It is Double Blinded as a matter of fact.
"Official Title: A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging, Safety and Efficacy Study of Oral ELND-005 (AZD-103) in Alzheimer's Disease."
Dr. Cruz speaks about it in reply to questions on the Quarterly Conference Call https://www.transitiontherapeutics.com/ir/webcast/Q1-2008.wav ....Q&A starts at 13:30 and goes for 14 minutes.
I think what you are getting at is that they can decide to start a Phaselll if they see efficacy through what is commonly referred to as "The Third Party Peek". Where the Trial stays Blinded but interim looks of 3, 6, 9, 12 months are performed by qualified unbiased persons (from FDA??) and the data is studied to look for Signs of Efficacy as compared to Placebo in the different Cohorts. If Signs of Efficacy is found then they can then decide to start to move forward with a Phaselll . It could be just in one of the Cohorts or all of them. One Cohort may respond more rapidly than another. Discussions with the FDA will help design the Phaselll Trial or the decision of smaller side studys.
This is where the Fast Track Status Benefits start to surface. It will allow the Trials to be given a lot of ability to be split up into many different smaller studys and Arms, Groups, and Cohorts
without having to declare a whole new Phasella or Phasellb or Phasellc Design etc. etc. but all the smaller studys will be combined into a common statistical data base. The FDA plays a very large role in these decisions.
To show Efficacy in the Phasell will pave the way to a Rapid Pivotal Trial and could even turn the current Phasell into a Pivotal Phasell/lll.
GPP All