The P3 format agreed with the FDA is simple. As per the company...

The P3 format agreed with the FDA is simple. As per the company announcement below endpoints and RSBQ have been agreed for one active dose group for a period of 6 months. To me we need to repeat what was already successful in P2 with a more accurate dosing regimen based on weight. IMO the success rates quoted in the above posts are very conservative.

"Neuren will conduct a single pivotal Phase 3 trial, using the Rett Syndrome Behaviour Questionnaire (RSBQ) and the Clinical Global Impression of Improvement (CGI-I) as co-primary efficacy endpoints. The double-blind, randomized, placebo-controlled trial will test one active dose group with a treatment duration of 6 months. The dosing regimen has been designed to achieve consistent drug exposure in subjects regardless of their weight. Neuren Executive Chairman Richard Treagus commented: “We are pleased to have held a very constructive meeting with the FDA Division of Neurology Products. It has provided necessary confirmation on the key issues relating to our proposed Phase 3 trial in Rett syndrome. We are now able to progress the final stages of development with full confidence. "