Full day clinic visit today. 11 blood samples, all the paper and physical tests, a urine test I managed not to fail, and an appointment to see Dr Brefel.
Where finally I got to see my datscans from the trial.
A trial for which I had to sign 3 consent changes. Partly because of covid19 precautions. Mostly because they have changed the primary objective after more than 2 years, just before the trial is due to end in March 2021
The primary objective is no longer safety and tolerability. Now, the primary objective of the study is to evaluate the clinical efficacy of BIIB054 via dose response using the change from baseline in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Total Score.
That sounds encouraging to me. I have no scientific or medical background at all, but I've always wondered why efficacy is only evaluated after safety has been established. If you're giving stuff to people, you might as well see if it works. I totally understand that Phase 3 studies are needed with a much bigger sample size, but it still seems like efficacy should be evaluated earlier. Good luck and please keep us updated. 😊
I suppose that's possible, but obviously they would have just stopped if the adverse affects were too frequent or too serious. All in all, I think it is more likely a positive sign, but time will tell.
I forgot to mention. My datscans showed no change. My neurologist told me that's normal and to be expected. You won't see any change over 6 months or even a year.
Fine. So why do one every 6months on the trial if it's normal for there to be no change?
michaeljfox.org/news/ask-md... suggests that there are significant differences in the first 4 years and that trials are using them to monitor effect. My next datscan, which I will try to see, is on Thursday 17th, which will be 30 months after the first one which confirmed my original diagnosis.
Sorry! I wanted to know if they administer some type of contrast medium through an IV prior to the scan. I haven't had one, so haven't researched them.
Yes. There is a radioactive tracer, and so i take potassium iodide on the eve of the injection, the morning of the injection and the following day to protect my thyroid
Are you saying they tell you the results of your DaTscans? I'm nearing the end of a 3yr Sanofi trial for a new drug called venglustat and they won't breathe a whisper on mine. And they won't promise to release them at the end either...I keep asking.
If I could add a little huffy emoji here, I would! :o)
It was like pulling teeth. I could only be shown them by my consultant neurologist and they kept failing to arrange it. I don't think they would do it routinely. And i am a bit of a celebrity as the Englishman in FranceHope your trial is a successful one. Thank you for participating
So, what do YOU think? Do you get any sense that your progression is slower than average?
BTW, when I began my trial I struggled with a choice between the study you are in or the one I am in. They both sounded quite promising. In the end I chose mine only because it happens to be specific to my genetics (I carry the GBA mutation).
I was already thinking: if could leap into yours next...or another similar antibody-to-alpha-syn-trial, I would!
I'm puzzled that there could be any other conclusion. My team were playing it cool - lots of Gallic shrugs and "we don't know, we'll have to wait and see"This time though, unlike the previous revisions I've signed, it's public published information.
So why hasn't the Biogen share price reacted? (There was a one day spike about the time the revision was published, but that makes even less sense)
Personally, I'm confused. I could have been on placebo last year and low dose this year, so would expect no benefit. To add to the confusion, i missed 2 infusions due to lockdown, started, stopped and restarted pramipexole due to a cough, severe breathing difficulties and heavy fatigue (which have now gone and not returned after restarting pramipexole, and may well have been covid19), and have tried 3 times to take B1.
My PD is unambiguously progressed from diagnosis, whatever the datscans show.
But
It's pretty mild. Dr Brefel asked the same question after examining me and concluded that my PD on Friday was almost undetectable (i was at my very best during her exam. It was more evident later in the day for the trial tests). I had not taken my pramipexole that morning, as instructed by the trial, so it was 28 hours since the last dose. I admitted I was not really too badly troubled by the pd, and that although I wanted to stay on the pramipexole, I wouldn't be that bothered if i couldn't. And i agreed that was different from 12 months ago, when I had a real urgency to start a symptomatic treatment.
I think I was on placebo year 1 and this year I'm not (that's to say - it's different this year). And maybe, although I'm progressed from diagnosis, and that's how I think of myself, maybe , ok probably, I'm better than a year ago.
That sounds VERY promising, to me! I would say my symptoms have progressed, but I think unusually slowly. I'm still one-sided. I still don't require dopamine, and I still think no one can tell...unless I'm nervous and then I get shaky.
I'm pretty sure I was NOT on the placebo.
But I just started on Neupro, 4mg patch...for restless legs at night. I'm pretty certain that's PD related.
I also eat a super strict diet and exercise a lot...so there's that confounding issue.
Sounds good. It is so hard to know what "normal" progress would be - in this "snowflake" disease. The neupro is a 24/7 dopamine agonist so will be working similar to my extended release Pramipexole. How long ago were you diagnosed? and how long were you on the trial for?
My normal trial ends in January but I have a 2 month extension to March - when the last entrant finishes their 2 years
A snowflake disease...right. So who can know? I was diagnosed in June of 2015.
It is/was a three year trial that for me ends this coming February. I'm actually on the warpath for a new trial now...but I'm having trouble finding one, and you might too. If you are far (year-wise) into your diagnosis you are disqualified from "early" studies, but if you are still phase 1 you are disqualified from "late" studies.
Thanks. Obviously I hope the drug is proven to significantly slow progress . Then we have to hope they have enough data from a phase 2 trial with over 300 participants to get early fda / ema approval. Fingers crossed
I wonder if the change in the success criteria is linked to biogens alzheimer drug which has got so much publicity. I doubt they really want to go through a phase 3 again with the effacey in doubt
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