Story about Trof trial, page-4

I'd take the particular claims - "Some of them even had their voices come back" - with a huge dose of salt. But your wider point is valid: positive word of mouth is likely driving a surge in enrollment.

It seems to me that the trial has morphed. Initially it was about safety and side effects, with efficacy necessarily a secondary concern in a phase 2 trial. But the additional enrollments are all at the highest dose (or placebo). That suggests safety is no longer a significant concern. The trial is double-blind, but safety and toxicity would be monitored throughout by a separate committee. This committee must have given the green light for maximum-dose v placebo for all new enrollments. And that suggest the objective of the extension is really about efficacy, to give the best possible chance to record a statistically significant difference between drug and placebo.

This is important. I'd imagine there would be a significant placebo effect in a trial like this; the parents invest so much hope in a trial like this. So congratulations to the company for being agile and flexible enough to extend the trial in such a way that gives it the best chance of demonstrating efficacy.

Doesn't mean the drug works, but they're giving it the best chance to demonstrate if it does or not.

GLTAH