Enrolling patients earlier in the course of the disease progression
I am surprised that the PPS went down a bit yesterday on the news about the protocol change with respect to enrolling patients into the trials with better vision to start with.
I see this as a big positive:
1) easier to find larger numbers of patients with earlier stage disease who may be healthy otherwise overall, and therefore, would not get rejected by other exclusion criteria.
2) greater chance that pts with earlier disease may have higher likelihood for demonstrating a measureable improvement in vision.
3) This is a show of confidence that the scientists at ACTC have that the procedure is safe to use on earlier stage disease patients who might not want to risk further visual loss with an experimental procedure. It will be excellent when we hear news that the IRBs at each clinical site have approved the change as well. And of course the FDA, hopefully Langer is helpful with that.
Check NCT01345006 on "clinicaltrialsdotgov" website
Sub-retinal Transplantation of hESC Derived RPE(MA09-hRPE)Cells in Patients With Stargardt's Macular Dystrophy
Study Start Date: April 2011
Estimated Study Completion Date:
Estimated Primary Completion Date:
July 2013 (Final data collection date for primary outcome measure)