DLP wrote:My mom was diagnosed with the early stages of AD a little over a year ago. She enrolled in Biogen’s aducanumab trial, but before she could take the last medical test to qualify, in March of this year literally days before she was to begin the trial, Biogen canceled the trial due to a statistical futility analysis. Upon learning that Biogen took another look at the data and now has decided to seek approval of the drug from the FDA, we contacted my mom’s trial coordinator to try to begin taking the drug, but were told that only those who actually began receiving the drug already would be permitted to have access to it while Biogen applies for and awaits FDA approval. We are heartbroken, as we understand this process could take years, and my mom can’t afford to wait years.
Please accept my deepest sympathy for your mother's dilemma. In 2017 I was one day away from starting the Generations 1 prevention trial when I developed an acute eye condition that they viewed as not being "medically stable." By the time I was cleared to start on the baseline 30 days later, the study had been temporarily halted while the Site's Institutional Review Board considered some changes to the protocol by Novartis. Because I had gone through all of the screening but was not yet taking the drug, I was not "enrolled" in the trial--the same situation your mother is in.
Worse, they could not use any of my screening because of a 90-day window between the start of screening and the baseline date (The screening window was later extended to 6 months.) It was a very long 4 months between cancellation and re-start, and I was truly depressed--even without a diagnosis of early AD.
Your mother was enrolled in the Screening phase, not in the Trial itself, so she's not considered one of Biogen's "participants". Biogen is apparently going to argue that it was a mistake to end the trial, because it has met one of the primary outcome points. They would not be starting a new trial; simply giving aducanumab (without a placebo "arm") to those they previously had in the trial.
Enrolling your mother would require her to undergo new screening. It's possible they could open up the whole trial again, including to new participants for another 18 months, but my guess is they simply want to get more people to the finish line of the previous trial and then the drug approved quickly.
I would guess that Biogen is going to be starting with low-hanging fruit and then pushing hard for more, something like this:
1) Early in 2020: Request for permission to re-start the drug/placebo in those previously enrolled who want to continue. This gives them and the FDA more data to analyze results. (My assumption is that the study sites still don't know who was on the placebo and who was on the drug, so it is still a "blind" study.)
2) Also early 2020: Biogen says it plans to request permission to market the drug. At this point, the FDA may come back with a proposal to add more people
to the study before marketing, especially if they want safety outcomes for ARIA as a side effect. Your mother could likely re-screen at that point. They already know she's motivated, has a study partner and has successfully completed testing. Keep in touch with the study site to be sure they know to contact you if your mom can be re-screened for enrollment.
3) it's possible the data will be compelling enough to fast-track approval. If so, you can bet that Biogen will get this to market ASAP.
Here's a link to an earlier post of mine from today about BAN2401, another Biogen immunotherapy drug targeted to those with MCI or early AD. They are in early stages of a large Phase 3 trial in 143 locations, and may have a location near your mother's: https://www.apoe4.info/forums/viewtopic.php?f=4&t=6709&start=10