Hi all,I would like to post same article third time regrading...

Hi all,

I would like to post same article third time regrading "failed CHF phase 2 trail" by using prof's own words.

The point about endpoints

“Primary endpoint” is a sensitive term at Mesoblast’s Collins Street HQ after a 159-patient trial of Revascor for end-stage patients using left ventricle assist devies (LVADs or heart pumps) came a cropper last year. Well, in the eyes of the market it did, because the shares tumbled 28 percent. In short, the trial - carried out by independent investigators at New York’s Mt Sinai School of Medicine and funded by the US Government National Institutes of Health - did not meet its primary endpoint of temporarily weaning patients from the LVADs. But Prof Itescu stresses the “academic” endpoint was set by the investigators - not Mesoblast - and was never viewed by the FDA as clinically relevant. “That [weaning] was not something of any interest to us,” he says. “What we were interested in, based on FDA guidance in writing, was reducing the major clinically meaningful problem of recurrent hospitalizations from major gastrointestinal bleeding.” “And we did. We reduced bleeding rates by 76 percent and hospitalization by 65 percent and these numbers were identical to an earlier pilot trial.” Mesoblast is now using an “innovative” endpoint that measures hospitalizations - a measure not targeted in early stage heart patient trials because thousands of patients are required to show a statistically valid result. Under what’s known as ‘joint frailty’ model commonly used in cancer patients, the total burden of the disease is taken into account. The secondary endpoint - the time to and incidence of mortalities - is simple enough. The ideal data package will result in reduced deaths and hospitalizations, but not reduced hospitalizations because more of the patients are dying.

Have a great weekend everyone!