total functional capacity

Just posted on the YMB by tdeank1, very informative.


TFC...let's dig deeper!

Total Functional Capacity (TFC) Scale details level of function in the domains of workplace, finances, domestic chores, activities of daily living and requirements for unskilled or skilled care...

In a study published in Neurology [2000 Jan 25;54(2):452-8], the annual rate of decline for 575 patients with baseline TFC scores of 7 to 13 was 0.97…

the mean baseline score across all three groups in the phase II HD trial was around 9…thus, they fall in the range of the aforementioned study published in Neurology....

the HD trial was 34 weeks, thus, you would then assume a decline of about .63 on the TFC…I did this by just taking the expected .97 drop that would be expected in a year, and prorated it by the 34 week trial duration...

WHAT DID WE SEE in our HD trial….250mg PBT2 = -0.3; 100mg PBT2 = -0.5; placebo= -0.6….

WE SEE THE decline in the placebo group being about exactly what we would expect [-.6 versus -.63]…

BUT…and here in lies the beauty…we see the decline about half that in the 250mg group [-.3]…when this is properly powered in phase III we should have statistical significance on this very important dependent variable