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- August 29, 2018 at 7:42 pm #7528
I’m surprised that I haven’t seen more about this discussed here…
The U.S. trials for pitolisant are underway. I’m fortunate enough to be participating as a patient. Here are details which may be of interest. I was not required to sign any kind of non-disclosure agreement (I did ask about whether one was required). Therefore, nothing I’m posting here is supposed to be secret.
The program is an “Expanded Access Program” rather than a true trial. No placebos are involved; all participants get the actual medicine.
25 centers are now up and running across the country (see here under “locations”); my understanding is that up to 40 are authorized. Because of internal bureaucracy, not every center has started at the same time. Up to 10 patients will be allowed per center.
Participants receive no monetary compensation, but are promised a free 2-year supply of the medicine (unless the FDA approves it for market before then, at which time you will have to get it through regular channels).
Getting into the program was not particularly hard. The full criteria are here. A previous diagnosis with narcolepsy with or without cataplexy is required. (I didn’t ask, but it looks like they are not taking IH patients.) The local center already had my medical records because my current sleep doctor practices at that hospital. A research assistant interviewed me once and that was it. They seemed quite eager to have me. You need to disclose all your current meds; some are forbidden while in the program. They have been very responsive to questions in this regard.
They phase in the medicine over time. Week one is 8.9mg, week two is 17.8mg, and week three onwards is the full 35.6mg dose. You take the recommended dose once per day upon waking in the morning. You are not required to keep logs or even to report regularly, so if you’re bad at that sort of thing, don’t worry about it.
They say not to expect to notice any effect until at least the one month mark and possibly as long as two months. I’m on week two of the phase-in (i.e. half of the full dose). As expected, I have not noted any clear positive effect yet.
I had cardiovascular issues with amphetamines and the Vigils, so I have been watching my blood pressure and pulse since starting on the pitolisant (on my own initiative; this is not a program requirement). So far, so good: blood pressure and pulse are in the normal range.
The only side effect that I have had so far is waking up for extended periods on a few nights: the very first night, one night after I took the pill too late (noon), and a seemingly random night during week two. It also has been harder, but not impossible, to fall asleep for voluntary daytime naps.
I hope to post with good news later.
P.S. I noticed after my initial post that Harmony now has a website for interested participants:August 29, 2018 at 8:59 pm #7530
Good for you! I wish you success!
It’s about time they got to the trials… it’s been a long time coming and I guess we got tired of following the development and waiting.August 29, 2018 at 9:13 pm #7532
…it’s been a long time coming and I guess we got tired of following the development and waiting.
I’m surprised that we haven’t seen more comments from European pitolisant users, whether here or on other sites, given that it has been available over there for more than a year…August 29, 2018 at 9:18 pm #7534
@tcpwn Thanks for the information! I stickied your post so hopefully more folks will see it. Please keep us updated! Although, I’m guessing you won’t know if you received the placebo or the actual drug until after it’s over I assume?August 29, 2018 at 9:19 pm #7537
Participant @tcpwnAugust 29, 2018 at 9:28 pm #7540
@tcpwn Ahh damn no clue how I missed that one! It’s an exciting medication at any rate since it works in a totally unique way compared to the other options. I also wonder why I’ve never heard of any reports from our European PWN. I wonder if it’s not often prescribed. I can imagine insurance over here requiring you to try other options before taking it since it’s probably going to be pricey until a generic pops up. I think Nuvigil was around $1,600 before it went generic.
I saw it’s on the fast track program for the FDA. Hopefully it’s effective for you! If it’s anything like the other medications, dosing can have pretty dramatic effects. 20mg and adderall XR was like a sugar pill for me. 30mg was amazing for a while.August 29, 2018 at 11:20 pm #7544
Choices. It’s all about choices because not everything works for everyone. I sincerely hope that it’s priced reasonably for those who need it. And, it appears to work on cataplexy as well. Bonus!
https://www.narcolepsy.org.uk/blog/new-study-published-lancet-neurology-shows-efficacy-pitolisant-wakix%C2%AE-cataplexy-patientsAugust 30, 2018 at 9:57 am #7546
I’ve actually inquired about this program (the website ‘peace program’ you’ve linked) in your post. I asked my sleep doctor about it because I was interested in becoming a participant when nothing else was working for me and my insurance wasn’t on the list of participating centers. I think he has to do something (from what I read) to inquire or get authorization to be able to add me or approve me to be on the list, but we are going to talk more about it at my next appointment. As of right now, I’ve found something that is actually having me feel a bit more myself, so when the time comes for my appointment your information and the additional research I do will be helpful for options to discuss. Thanks!
-JohnnaAugust 30, 2018 at 2:53 pm #7551
Thanks for posting that you are participating in the trials. I hope that you will have good results! When you have the time, please update us as to your positive and/or negative experiences along the way.
As @ferret pointed out, “Choices. It’s all about choices because not everything works for everyone.” I agree that it will be good to have another option available and an added “bonus” if it works for cataplexy.
@tinyelephants I’m glad that you are continuing to feel better! Keep us posted.
@jasonm Collaborative information…just one of the many things that I appreciate about these forums… 🙂
It will be interesting to continue to follow this closely. Here’s a quick link to the other posts related to pitolisant from a search on this site. Please keep the links and information coming. Thanks!August 31, 2018 at 2:23 pm #7599
@tcpwn Ahh damn no clue how I missed that one!
You were probably sleepy!
my insurance wasn’t on the list of participating centers.
Did you mean your hospital/clinic wasn’t on the list? Insurance has nothing to do with it. As noted above, participants get the meds for free. They didn’t even take my insurance information.
When you have the time, please update us as to your positive and/or negative experiences along the way.
Yesterday and today have been excellent days, so I’m thinking that the effects are kicking in. Early days but hopeful…August 31, 2018 at 10:31 pm #7602
@sleepy-rbd, thank you very much 😊
@tcpwn, oops 😬 yes, that’s exactly what I meant. Apologies. Glad to read you’re having a first few excellent days, hoping for many more for you, please keep us posted along the way when you can if you don’t mind.
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