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Re: imho post# 169026

Saturday, 10/20/2018 9:26:00 PM

Saturday, October 20, 2018 9:26:00 PM

Post# of 518980
My guess is the 15 person trial will be open label since it is a safety and tolerability study. There is no need for a placebo group.

My take is that the 15 person trial is to demonstrate that the Phase 1 results are valid for the Rett girl population and if they are not, it will determine the MTD for the girls as well as test for safety.

Any efficacy demonstrated will be a bonus.

If there is ANY efficacy demonstrated recruitment for the larger trial will be pretty quick since there is not much out there for Rett girls that does much good. Remember that 2-73 has quite minor side effects compared to the drug the Rett girls are presently taking. That is going to be a big draw for the trial.




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