[www.prnewswire.com]

The long awaited European Isis-htt trial looks to have begun. I was interested to see that it is now a stage 1/2a trial. Initially we were told that it would be a stage 1 trial just for safety but they seem to be also looking at it's initial effects on symptoms.

This is the first drug of it's kind - the first to attack HD at the roots. Any thoughts /comments Marsha?

Kathy x

Awesome news.... PRAYERS THAT THIS IS THE BIG ONE !!!

Such excitement with HD in all!!! But this one is the one we've been waiting for!! BIG PRAYERS!!!

Yes, I originally read it would be stage 1 trial, I wonder what a stage 1/2a trial means

I don't understand how something like this can be put out by isis, with no info on the clinical trials, I've tried to find something somewhere online about the trials, who's recruiting, who qualifies, everything like that, and I cant find anything. And I don't know what the dif is between a phase 1 trial and a phase 1/2a trial is. It was originally supposed to take place in Europe and Canada, but I can't find anything. The ubc website has nothing about it. I will have to take a look at the hsc website, maybe they have something. Now the hsc might have put something out in a newsletter, I don't read all the newsletters, if they have then that's great, but if they haven't, I will request them too. That is actually a very effective way to let everyone know about trials. Just like in the US, you guys wonder if people know about trials, because a lot of hd patients don't go to doctors, well a newsletter from the hdsa would also be a very effective way to get the news out, and maybe they've already done this. Months ago I was able to find a write up about where the trials would be, and who would qualify, now I can't find anything.

Marsha, do you know anything, please help if you're able to, we still need you tons

Professor Tabrizi at UCL is very involved with this trial. You can find some information here

[hdresearch.ucl.ac.uk]

In the interview she gives she stresses that the initial trial will be solely for safety but some articles I have read this week in the UK state that they are now also looking at the effect of the drug on trial participants. That is why is is now called a 2a trial as well.

I still think that the number of trial participants will be very small and possibly hand picked. Hopefully if the drug goes to a full stage 2 trial more participants will be studied and people will have chance to apply to participate.

One thing they have stressed in the past though is that, initially, if this drug reaches the market it will be for non symptomatic & early onset only.

Kathy s

There is no way they can hand pick people. This trial is a small one, but it is supposed to take place in Europe and Canada, and it is supposed to take place here at ubc in Vancouver. I cannot find anything online, or on the ubc website about the trial. I guess what I'm saying is first I'm so glad you posted this, I've been waiting to find out when it starts. What makes me mad is, here an article like this is put out, and people like us, the people with hd, have to spend hours googling trying to find out any info at all, on where the trial sites are, what the qualifications are, etc etc etc, and this is making me so mad, that the info is not getting to us in an easy way, and it certainly would have been nice for the article to explain what they meant by saying it is now a plase 1 2/a trial, what does that mean, you know, I guess im feeling really mad

Marsha, please, if you know anything about the trials, could you please post

PS Im not mad at anyone here. I'm so glad this article was found and posted, thank you. I'm mad that you, me, marsha, anyone and everyone here, that we all have to always do the digging around the internet, to try and find out info that we need. It's not fair. It's not right that they put out an article like that, with no further info for any of us. Now I guess I now need to find out if the HSC knows anything, and then I guess i'll have to phone ubc, but none of us should have to do this, but we do do this, because if we didn't all do this it wouldn't get done. I also know that robi from chdi probably has this info for us, so if you have the info on the trials that we are looking for, then please post it robi



Edited 1 time(s). Last edit at 07/26/2015 03:20PM by Barb.

Well, I just emailed Robi, he should be able to give us some info, ANY info would be very much appreciated, even the HSC has nothing on their website



Edited 1 time(s). Last edit at 07/26/2015 10:08PM by Barb.

Hi Barb, and others!

Thanks for asking me to comment. I agree with your feeling that the announcement of the Isis antisense trial is exciting news. At this point the conduct of the trial is in the hands of Isis and they are responsible for disseminating information. As I am sure you are aware, once a trial is about to start what the sponsor (in this case Isis) may say to potential participants becomes more regulated by Institutional Review Boards in order to ensure that peoples’ decision to participate is properly informed and not coerced or pressured. Although I don’t know for sure, that may explain some of the limitation on what you are hearing or reading directly from Isis.

That said, let me suggest a few things:

1. You are correct that the University of British Columbia will be one of the sites; Dr Blair Leavitt is the investigator at that site.. In fact, this story was already published about that: [metronews.ca]. The trial will also be taking place at a few sites in the UK and Germany. Dr Sarah Tabrizi at University Collegte London is the principal investigator of the trial.

2. “Gene Veritas” lives not far from Isis’s headquarters in Carlsbad, California and has covered the program quite extensively in his blog. This is his latest post: [www.curehd.blogspot.com]. It also references earlier posts.

3. It is my understanding that the trial will be posted on clinicaltrials.gov sometime in the next few weeks. I will post here on the message board as soon as I know that happened so everyone can have a look.

4. People should feel free to post questions here on this message board. I will watch out for them and make sure they are passed on to Isis. Last I heard the company was working on a “Frequently Asked Questions” type of document. I am sure they would value input into that process from all of you.

[Barb- I took the liberty of incorporating some of my earlier email to you. I hope that's OK.]

Regards,

Robi

Robi Blumenstein
CHDI Management/CHDI Foundation
robi.blumenstein@chdifoundation.org
www.chdifoundation.org

At the HDSA conference, there were a lot of people asking about the trial. Basically the ISIS reps that were there, let everyone know that the study has already started. It is taking place in both Europe and Canada. All of the participants are already picked out, and it is less than 40 people that will be in the study. There is no way for anyone to become eligible the next phase (if it is safe), for probably at least two more years. The first year of the study will be mostly focused on if it is safe and what are the best doses if it is safe. There will also be no info being posted because of all of the trade secrets that have to be kept private. Unfortunately the only way any info will come out early, is if it is lethal and they have to stop the study. I think that this Q/A session was recorded and posted on the HDSA website. It was during the Saturday luncheon period. But I don't know if it was posted online or not. That's about all I know. There might be a little more accurate info on those conference videos.

Thank you Robi for posting, and for letting us know when it will be listed on the trial site. And Brent, thank you also, that definitely explains a lot, but also raises more questions. I guess as long as I've been around the forum, I've never heard of participants being hand picked, I'm kind of shocked. It seems really strange, because usually trials let people know when they are recruiting, so people can choose if they might want to participate. So I guess I'm wondering if this is how the beginning of most trials go, and then later they start recruiting, and I just didn't know that, or if this hand picking is something new, a first. And then how did they hand pick, I don't get it. I've just never seen anything like this before, and I'm wondering if this is the norm, and I just didn't know it, or what the deal is. This is very confusing for sure, well to me anyways, don't know if anyone else sees it this way, but again thank you

Apparently the places that are actually doing the studies, wanted patients that they have known for a while and had a pretty steady history of their condition. Plus they needed people that were for the most part pre symptomatic, that are able to communicate finite changes in how they are feeling. That way if there is a dramatic situation that comes up, it will be much easier to notice changes from people they know well. Whether it's a good or bad reaction to the drug. They honestly don't even know if this drug isn't extremely harmful to a human. Also this is not an anti chorea or a symptom reversal medication, which is why they didn't want very advanced staged pHD's. But if all goes well and they make it to stage two trials, then the recruiting will be opened up to everyone else. But remember that the stage two trials are still at least a minimum of couple years away. Possibly even longer.

Thanks Brent, but now that's even more clear than mud. A year ago, I had no problem finding the trial criteria online, and it said the criteria was early stage, with chorea, so they could use the chorea to judge how the stuff was working. Well, I'm early stage, with no chorea, so I didn't look into it, because from what I read I would not have qualified.. That info was easily found a year ago, now there is nothing online about the criteria, and according to what you've just said, and the criteria has changed now, so now I would have qualified. So great. I am very early stage, so much so that my scores are very low, and sometimes hardly no score at all, oh yeah, and I've been like this for 11 years. I'm mad at how all this info has not been properly available, for those that would have been interested, or to at least in the very least, when they made their big announcement that trials had begun, to have the consideration to let people know that recruiting would not be open for a couple of years, if that's what the deal is. That is what I would have called considerate.



Edited 1 time(s). Last edit at 07/29/2015 05:48PM by Barb.

Hi Barb,

Yes, I understand your frustration! As a person who has participated in several clinical trials and research studies, I was really hoping to be considered for the Isis trial. I am 60 years old, with a CAG of 42, and don't really have any symptoms yet (I think). So, if they were looking for a wide variety of subjects, why not you and me?!?!

But I live near Chicago, so if distance is a consideration for Isis, maybe that would rule me out. But what about you? You live in Canada! You should be a shoe-in!!

I agree that it is difficult to find information about any studies on the internet. The studies that I did participate in were because I was already enrolled in Predict HD at the University of Iowa. I think Clinicaltrials.gov is very hard to use, as well.

Judy Galasek

They said that the study participants needed to live within like a hundred miles of the test sites for emergency situations. They didn't say exactly which criteria was used. Just some of the general things that I mentioned earlier. But there was apparently a huge amount of disqualifiers for the phase 1 part of the trial. The participants for the trial were already picked before the conference. And they were starting it in July. That's about as much info as they were able to give out. And I know how you feel about the lack of info as well. A lot of people wanted to be a part of the phase 1 trial. Which is why I think they went the route that they did as far a selecting the participants.

Sorry to be so late chiming in. Thank you, Robi, for sharing information.

I am very excited about the potential of the trial.

I haven't heard or read that if it proves to be a treatment that it would be withheld from all but the nonsymptomatic or those in the early stage of the disease. That doesn't make sense to me. Robi, can you address this?

So yesterday the HSC facebook page did a post, about this trial, and that patients in Vancouver are beginning the med this month. Marsha, I have not read the article, only read the headline, because my concussion is really bad again, went on a bus ride yesterday and the bus driver was slamming the brakes on, and I'm paying for it now. So what I'm saying is I'm not up to reading the article at all today, but will when I'm up to it.



Edited 1 time(s). Last edit at 08/13/2015 04:18AM by Barb.

So I have finally been up to reading the link I posted in my above post. Wow, I had no idea what I truly posted. Anyways, the low down on the article, is the globe and mail did a story about the trial, and about hd, and it said that 6 people in Vancouver have been picked and are beginning the trial in august. It also said these people all had to live very close to ubc, because they would need very close monitoring for 6 months. It was a very good article. I've decided to delete my above post, just because a summary is all that is needed, and because there are some things that I didn't know, that maybe only angels need to know. Yes, I'm talking strange, that's ok, I have hd, and we're getting closer to a cure