New Clinical Trial of Investigational Study Drug Recruiting in the US and Abroad Eli Lilly and Company has opened a large, multi–center phase 3 clinical trial to determine if an investigational study drug can slow the decline in walking ability of boys who have Duchenne Muscular Dystrophy. The study will also assess the safety of this investigational study drug and any side effects that might be associated with it in boys who have Duchenne.
Enrollment Criteria include:
Males with Duchenne Age 7 – 14 years old Ambulatory (walking) Receiving systemic corticosteroid therapy for at least 6 months prior to screening Have a left ventricular ejection fraction (LVEF) ≥50%, as determined by echocardiogram Participants may be eligible for enrollment regardless of their specific genetic mutation.
There are currently sites in 17 states in the US, as well as multiple sites in 14 other countries (Asia, Canada, Europe and South America).
Accelerated Approval = AA based on what?
Can FDA change regulatory process on for SRPT alone ?
What is the gaurantee that FDA will accept NDA Filling by end of 2014? If YES then BASED ON WHAT? 2 Patents out of total 12 undertrials performed well - Based on what? If trials were started in 2007 until 2014 -Why P2 data is not released or Primary End Point ?
Meeting with FDA to put pressure - Why? There are many other options available for FDA to approve other than SRPT and actually those options are nearing completion of their Phase3 trials, whereas SRPT still hiding P2 and Promoisong it be starting soon. Where are they all these time? why the urgency now ?
If you add these Mathamatics PROBABLY YOUR ANSWER WOULD BE = ZERO
Good job and with great respect--I reverse engineered the data you presented --ran
everything through my Cray Computer to develop the formulas you used and
it all checks out----My Cray Computer did call my attention to the third component
suggesting a typo---should be 213 down from 215 (not 2015)---
Thanks for that nice quantitative analysis! I don't think I can improve upon it. However, I may add in a shortcut to the finish line which may compress the timeline: IF there is a 4th bx; and, IF it shows increasing 'density' of dystrophin, then AA should be assured rapidly with dystrophin as surrogate.