Hi Folks-

Here's the link to information about the Isis ASO trial on ClinicalTrials.gov:

[www.clinicaltrials.gov].

Robi

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

Thank you!

Yes, thank you Robi. They actually had dr hayden and my neuro dr blair on global bc news two nights ago, and did an announcement about this trial being the first possible cure for hd, yes, they used the cure word. Me and Ray watched the announcement together, and I thought, I got to see this announcement in my lifetime, yes, and I got to share watching it with someone I love, nothing better than that. Yes I know, we don't know the results yet, and know this will take a lot of refining probably, but still, to hear the announcement, first possible cure, and to have witnessed that. My dad would have LOVED to have heard that!!! But I know he witnessed it in heaven

So I just checked out the trial link Robi gave, it's a very good link, everything I wanted to know about the trial, and about physical health criteria to get into the trial, etc, the things I wanted to know. It also said they are not doing recruiting yet, and I think that is true and false, because it turns out they are not really doing recruiting, but they are doing hand picking. I know this because global bc did a quick blurb about the trial two nights ago, actually very quick blurb, but it was very exciting to see on tv. They said clinical trial at ubc has begun for first possible cure for hd, yes, that's what they said. And they also said that 6 patients have already been picked for the trial, and the trial is beginning now, in august. I think the reason, my own opinion, that hand picking has been done, is because it is only 6 people at ubc, and they prob know their patients best, as to who would meet all the criteria. I think because this is gene silencing, that is why they are cautiously starting with such a small group, for safety reasons, then I think I read somewhere that the next stage of the trial, if it proves safe, would be open to recruiting, and I think I read in the US too.

This is great news for everyone. It's bad news for me since I'm over the age limit to be a subject (65). However, the good news for me is that I just turned 69 and am still kicking!

Will

Hi,

Yesterday, I sent an email to the address provided on the ClinicalTrials.gov website, and actually reached a real person. The person I contacted was Lori Cooper (LCooper@isisph.com). Here is her response;

Thank you for reaching out. We are planning for a randomized placebo-controlled Phase 1/2a clinical study to evaluate the safety and tolerability of ISIS-HTTRx in patients in the early stages of HD. The study will be held at participating sites in Canada and Europe, and unfortunately this preliminary study will not have any sites in the USA at this time. I’m sorry to say that the study sites will not be accepting participants who need to travel more than 4 hours to get to a study site, and will not be accepting international travel participants if you are thinking of relocating.

This first clinical study is very small, safety study with only 6 sites participating in Canada and Europe. The sites will be recruiting approximately 36 patients total from already established patients that live within a limited distance from the site to accommodate the multiple study visits. The trial will be evaluating very early stage HD participants (known as 'stage 1' - typically within 1-2 years of symptom onset) so that any adverse effects can be easily detected from disease symptoms.

This clinical study will likely run for approximately 18 months. Recruitment is anticipated to start late August/September. Inclusion/exclusion criteria and study details are posted here on ClinicalTrials.gov. ClinicalTrials.gov Identifier: NCT02519036.

Please understand this is the first step for the clinical studies to ensure the drug is safe in humans. If it is proven safe, then additional, much larger clinical studies will be scheduled and we will have sites in the USA at that time.

Isis Pharmaceuticals and our partner Roche remain fully committed to developing this potential therapy and we are all hopeful for positive safety data from this first clinical study, as it is just the first step in the clinical development process.

I'm attaching an information sheet on ISIS-HTTRx to provide additional background on our drug. I hope you find it helpful.

Please let me know at any time if you have further questions.


Sincerely,

Lori
Lori Cooper
Patient Advocacy
Isis Pharmaceuticals, Inc.

E: lcooper@isisph.com

Ugh. 18 Months.

Thanks for sharing.

Marsha or Robi, would either of you know this, or have a good guess at this question, I'd really like to know. Giving best possible results for this trial, and further trials, I want to know a possible future. Would a possible future be that people with hd would need to get spinals at set intervals to stop the disease, or, are there ideas that this could eventually be turned into pill form???

Also, this will be silencing both hd genes, enough for a holiday, but I always thought just silencing the bad hd gene was what was hoped for. I realized that silencing both genes is what we have now, so we go with that, and that slowing just the bad one is maybe more difficult? So are we doing it this way just because the other way isn't developed yet, and that we still want to wait for that. Or is it more costly to silence just the bad one? Or maybe this is easier, if this does work, to do this first. I realize this is huge, and this is what we have now, but isn't silencing just the bad gene better? It's just my thoughts, and I don't understand if this is antisence or rna, and what the difference is between them, and if they have anything to do with what I'm asking

I may be clueless as to the most concentrated populations of people with HD, but I sure as heck don't think of Canada or Europe. If you want candidates who must be inside of 4 hour driving distance, why wouldn't you put one in... Indianapolis, for example... which draws from Chicago, St. Louis, Louisville, Cincinnati, for example?

You have to go where populations are where you have enough candidates, and candidates which are likely not to drop out of the trial. Are some of those European sites expecting people to cross country borders? It's easy to do that, but it can be an issue all the sudden if there were weird passport issues.

I'm selfish I suppose, but I am also of the opinion that there are more persons with HD in the USA than other areas, per capita. I must be wrong on that one.

Actually no, bc is 1/6,000, not 1/10,000 like the rest of Canada and the US. Never heard anything so foolish, where would these places find enough candidates that are closeby. They only need 6 each. I don't know why they chose the sites that were choses, but i'll tell you one thing, UBC is one of the top research centres in the world, why wouldn't you choose ubc. Sorry, find your post a bit foolish. I'm sorry, why am I responding, your whole post is so, just strange...hd per population??? huh??? I don't know anything about how trial sites are picked, but I am sure it is not by population. The hd clinic in Vancouver is first, doing some of the best research in the world, and is a leader in clinical trials. These trials are in Vancouver and Europe, at large well known, hd research centres. Why wouldn't the large research centres have a large data base of patients? I just don't even get your thinking. Maybe it makes sense to start it with dr Leavitt at ubc, because, oh yeah, didn't he just finish inventing a test, just a few months ago, testing spinal fluid, that tells if there is little improvement in a persons hd, oh yeah, that test will now be an important part of this clinical trial to tell how the patients hd is improving during the trial. Oh yeah, why pick Vancouver? Or Europe? I don't get it. Why the hell not pick Vancouver and Europe. I don't know how they pick their sites, but they have picked some very top notch research centres for sure.

PS And sorry, not trying to be offensive, and sorry if I was, I was just kind of stunned by your way of thinking, and maybe just don't quite understand your line of thinking. I wouldn't care where the trials are being done, just glad they are being done. And after safety is proven, like nobody dies from it, then it will be open at a lot more centres from what I've read. I'm glad they aren't trying their first experiment in this on a huge amount of people quite frankly, and am very anxious to see where this goes



Edited 1 time(s). Last edit at 08/21/2015 03:32AM by Barb.

I guess we can hope for the Midwest area in USA for phase II/III, which is fine. Barb, locale doesn't matter all that much, I just like the buzz it would create in local media and get some advocacy... anything... for visibility purposes. USA is probably top six or seven countries in the world for HD per capita and ISIS is a USA based company, but whatever.

I imagine that toxicity, efficacy, and knockdown endpoints have already been met to an extent, so I am not too worried about mortality, but that is why they have Phase I/II one would suppose. Should be safe it primates handled it but not sure.

Barb, I sense you are an angry elf because canucks haven't walked off with the Stanley Cup for over 20 years.

Lol Tyler, I don't watch hockey, or any sports of any kind lol Yeah, the first part of this trial is completely on safety, and I read after this, it will also be opened up to the US, if all goes well in the safety trial first. I get what you mean now too, think we're all just venting on what has not been a very transparent process in this trial. If I ever get a canucks jersey, i'll send it to you lol

Sorry Will!!

Your just going have to will it away! As you have!! I wonder if this is a FDA thing and easier to go elsewhere.

HOPE!! I try not to get TOO excited. There's a lot of failure in clinical trials.

Robi, what does "recent" mean as an exclusion. And have we had any good input from other trials, that is not official?

mare,

Thanks for the kind words. Here's a Runners World article about my next event, which I'm training for now.
[www.runnersworld.com] I guess we never got the word that it was time to stop. Like my battle with the HD gene.

I read a piece recently about a terribly wounded young Marine - he lost both legs and one arm. It doesn't bother him. He skis, sky dives, kayaks, cycles and does assisted "mud runs" with military folks. His personal advice for other people is: "Your excuses are irrelevant". I think of that when it's too hot or too cold or raining too hard to run.

Will