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konagirl

Active member
Joined
Oct 16, 2015
Messages
56
Reason
CALS
Diagnosis
04/2016
Country
US
State
FLORIDA
City
Jacksonville
Hello,

My husband is eligible for 3 different trials coming up in the next month or so under Mayo here in Jacksonville (Dr Boylan). Because they will all overlap on time frames, we have to chose just one. I'm curious to know, does anyone have any experience with the phase 1 trials? My gut is telling me to take this route but I'm just unsure. Our options are this:

A: GDC-0134 (Phase 1 study) "This is an oral medication that is thought to help by blocking the activity of a protein in your brain cells called DLK (dual leucune zipper kinase). Blocking DLK my protect neurons and prevent them from dying in ALS"
This will require 4 consecutive nights in the hospital 4 separate times, with the trial lasting between 3-5 months. They are accepting 32 patients and our Dr is slotted for 8 of them. The study is funded by Genentech.

B: Ezogabine (Phase 2 study) I believe most of you are familiar with this one. The study will last ~14 weeks and there will be 192 patients accepted, 12 of which where we are.

C: Tirasemtiv (Phase 3 study) I believe most of you are familiar with this one too. This study will take ~60 weeks to complete and they are accepting 445 people, 35 of them with our clinic.

Thoughts? Weigh-ins? My husband turned 37 in March, diagnosed with PLS in January of 2016 and just recently his diagnosis changed to ALS on April 14 2016. He is showing no bulbar symptoms, he has severe spasticity in his legs and his hands are losing strength quickly.
 
Only 4 snippets from me:
1. Boylan is great.
2. A phase 3 study would logically have greater chance of involving a successful therapy.
3. Sooner the better.
4. I've no expertise, no clue. I'm sure someone much more well-informed will come along with better advice.
 
I truly am with you on wanting to find a cure for this nasty disease. And clinical trials are going to be necessary for those that are coming behind our PALS in other generations. My PALS was hoping to do the Ezogabine but we live in MT and he would have to go and stay in SC with his brother and sister in law for the duration. Travel and cost of planes for us both, plus teen in school, etc. He was/is hoping to find something that is going to "fix" him. I had to explain that a clinical trial is just that. Even all the fine print says "even if it looks like the treatment is helping, upon completion everything is stopped" And that he also could fall into the Control group or the placebo group. We had to weigh the "time spent" along with "quality of life". I so wish there was something. And if we did live closer I believe we would do the trial just to see.
Take care.
Katie
 
I hear ya, I really do. I try to keep a fine line between hopeful and realistic. For my family, carrying on hope is a must for my husbands mental status. We have two small children also and while the 3 year old doesn't understand, our 8 year old benefits from seeing us try everything we can.

I had a lot of questions for our Drs clinical coordinator around that same point....even if we are getting benefits, you mean we have to walk away from it at the end of the trial?!? She said that the phase 3 company for Tirasemtiv - they have already agreed that if the studies are good they will allow the participants to remain in an open label phase to continue the drug. The phase 1 she was unsure of but did say it is possible. I am stuck between those two.
 
I'm currently screening for a clinical trial that requires lots of travel back and fourth to the research facility across the country. I thought I'd share that in this particular clinical trial, the drug company covers all my air travel, ground travel, hotels, and even $50 for meals a day. I was unaware that the drug company paid for everything in certain trials.

I don't have much knowledge of the 3 trials you mentioned, other than the phase 3 trial of Tirasemtiv sounds promising.
 
Dylan the phase 1 and phase 3 that I asked about both do that too! We are fortunate enough to have mayo right in our back yard so there no need to travel for us, but I found it very interesting.
 
Apologies for the late reply -- one of those middle of a post forgotten, log out nights. Perils of tabbed browsing.

GNE has an excellent R&D hit rate but likely even if it's effective, the dosing regimen will change at some point so clinical benefit is least assured in Phase I.

Ezo is on the market so if it proves helpful there will be a way to get it outside trials, indications or not, if you are willing to potentially pay the freight.

Tira's results thus far have been modest.

If you want to benefit science, of course, Phase I is the highest value. If you want to be most sure of getting some value (if you are in an active arm), then of course Phase III is the way, though not locked and loaded. AEDs (ezo) have enough neuro side effects at therapeutic dosages that I would characterize that as the trial most likely to bring you a tolerability or safety issue.

Best,
Laurie
 
Thanks Laurie, so much of what I read from you is always so helpful to everyone, I was hoping you would see this :) I am still learning a lot of this terminology, can you tell me what you mean by this....excellent R&D hit rate
 
Thank you for the kind words, KG.

Genentech (GNE) has a better "hit rate" than most in terms of looking into [deciding to proceed to Phase I] molecules that turn out to be safe and effective as opposed to not sufficiently safe and/or effective to continue. They also know how to get compounds to market, including "first in class" [of its kind] as this one is, whereas other companies don't always have the wherewithal to do that, e.g. negotiate with regulatory agencies, payors, etc.

So if this were "no-name company hoping to cash out their investment and live in Tahiti" or certain big pharmas I could name, we would have more concerns than with Genentech (disclaimer as for all major pharmas: a former client of mine). They are not perfect; no one is-- but a better bet than many.
 
Thanks for sharing your knowledge. We have a big decision to make tomorrow! My gut is telling me phase 1, we shall see.
 
I just left Mayo Jacksonville and Dr Boylan offered me inclusion in 1 of the 3 trails.
Just being diagnosed I am overwhelmed and kind of unsure of the next step. Of course I am fearful of adverse effects from a trial drug? Has anyone had adverse effects from a clinical trial drug? Plus have you made your decision on which to try? I truly need help I am overwhelmed!!!
Thanks. ��. Sorry if double posted
 
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Being part of a clinical trial is an important contribution to science so thank you for considering it. If you are concerned about side effects you might start by looking at how many patients have already taken the drug. Something in phase 3 likely has had more patient doses. If it is something like retigabine which is on the market for another condition they don't know what it does to ALS yet but they have a good idea of its safety protocol otherwise

If there are reports of earlier phase trials available read them. You may get an idea as to side effects or possible benefits

Look at the trial design- how many visits? How frequent?

I heard someone- I think it was Dr Bedlack ( if not it was another leading ALS doctor) -say once that PALS in clinical trials were shown to do better than average even though the trial drugs have failed efficacy. This could be patient population differences but he theorized there was benefit in the monitoring, interactions and relationships that happen in a trial. I believe this. I have only ever done observational studies but I think I have learned and benefited from participating
 
Hi Phyllise, have you met with Dr Boylan's clinical coordinator yet? Her name is Amelia and she is a wealth of knowledge. When we were weighing our options, she brought us in and thoroughly went through each trial with us in depth and answered all of our questions.

We are enrolled in the Phase 1 study of GDC-0134. Did the eligibility screening this past Tuesday (it took aaaaaalllll day) and he is scheduled to check in to the hospital on 6/6 for his first dosing. Because this drug has never been tested in humans, any possible side effects are very unknown, but Emory just dosed their first patient 2 weeks ago and he is still breathing, so I guess that is a plus. By the time we go in, several others will have been dosed by then and if there are any negative side effects we will be told.

This trial does require a lot of hospital time and follow up visits, but since we live right near the hospital this works ok for us. I see you live in TN, if you were to chose this trial I do believe the sponsor company reimburses for travel. I believe that goes for the Tirasemtiv trial as well, Amelia can confirm though.

It can be an overwhelming decision to make. After many many many hours of research, we ended up going with our gut feeling.
 
Yes I met with Dr Boylan for 2nd opinion and he confirmed my diagnosis. At that time he offered me the 3 trials.
The unknown is scary but the disease is as well?
I am on my way back to TN now and will have my husband call them back next week. My voice is getting worse.
Will you please stay in contact with me and I will with you. I have an ALS coordinator in TN that is trying to gather info also
Please let me know (good I certainly hope! Or bad)of anything you hear. Your husband will certainly get his dose before me if I get to do it.
I am new to the forum and still learning this reply thing but any responses come to my email.
It is great to know others that are searching for hope. I by no means am glad of the situations but of the support.
Thanks
 
Kona,

First, it's great that you live in Jacksonville and are that close to Boylan and Mayo! Prayers and hope that the clinical trial goes well.
 
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