Is the 15 patient study also described as double blind placebo controlled, or is that only describing the follow on study?
“Pharmacokinetic and dose finding will be investigated in a total of 15 patients over a 7-week treatment period including ANAVEX®2- 73-specific genomic precision medicine biomarkers. All patients who participate in the study will be eligible to receive ANAVEX®2-73 under a voluntary open label extension protocol. This study will be followed by a planned placebo-controlled safety and efficacy evaluation of ANAVEX®2-73 over a 3 month treatment period.
So the first part of the trial will probably be open label so patients can be closely monitored for signs of side effects in an effort to determine MTD for Rett patients. The inter-patient variability and the age related variability in drug clearance are probably why the FDA is requiring the 15 patient study first. If I recall correctly Anavex wanted to let parents administer A2-72 in-home rather than in a clinic , so it’s no surprise the FDA wanted to be cautious if that was the case.
Probably similar to the Alzheimer’s 2a two part design imho.
“This is a Phase 2a study consisting of two parts, PART A and PART B. The first part (PART A) is a simple randomised, open-label, 2-period, cross-over, adaptive design study lasting for each participant up to 36 days
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