My comment that there would be a 19-20 week assessment period per patient was based on the trial website that Acadia has set up
The LAVENDER study is a 12-week Phase 3 double-blind, randomized, placebo-controlled study evaluating the efficacy (how well it works) and safety of trofinetide, an investigational medication administered as an oral solution to treat Rett syndrome. Qualified individuals will participate in a screening period of up to three (3) weeks, a treatment period of twelve (12) weeks, and a safety follow-up period of thirty (30) days. During the treatment period half of the study participants will receive trofinetide and half will receive placebo. *
My assessment of 18 months from trial commencement to announcement of results doesn’t include the 40-week extension trial. The extension trial shouldn’t delay announcement of topline results for the parent trial but it could possibly delay NDA submission if Acadia wants to use interim data from the extension trial to enhance its Ph 3 data.
The company running out of money isn’t something I’m concerned about – I’m confident that some kind of transaction will happen before H2 next year.
I very much share your hope for positive Phase 3 results, otherwise, I wouldn’t be invested here. However, I also never consider clinical trial results or FDA approval to be a shoo-in. The last two links concern the Phase 3 trial of arbaclofen for Fragile-X. Despite widely reported, significant and positive changes in patients on the trial, the primary endpoint wasn’t met and the trial was abandoned.
(20min delay)