Clinical Trial Decisions

[konagirl]

Thanks Kim, we appreciate that for sure.

 

[scaredwifetx]

Steve has been in the Tirasemtiv (Phase 3 study) trial for three months and has had ALS progression. Of course it is a blind trial so he may not be receiving the actual drug. He also has hand no side effects either. I was thinking about cures or slow downs of the symptoms earlier tonight before reading this post and wondered why there has not been a drug invented to feed the muscles. I am not as educated on this disease as many CALS and PALS who are on here but if there was a trial based on that theory I would love for the chance for Steve to be in it even if a phase one trial.

 

[affected]

Deb what do you mean 'feed' the muscles?
The muscles die because the nerves activating them die - so it's not a muscle disease, but a nerve disease. So only something that can slow, stop or reverse nerve death will impact on ALS.

 

[phyllise]

I was wondering how the trial is going?
They said my thyroid cancer dx might eliminate me from the study but wanted to keep up with your husbands progress in the trial.
Thinking about you.

 

[konagirl]

Hi Phyllise,

Chad got his first dosing last Tues and had 0 side effects good or bad from the medicine itself. He went into the hospital 6/6, received the dosing 6/7 and was discharged 6/10. That was the hardest part...being in the hospital that long, along with the rigorous testing protocols.

Chad is still able to walk, he has severe spasticity that makes his walking difficult, but he is still able to do it. After laying in the hospital bed for a week, I do believe it inhibited muscle wasting. His walking has now severely declined and he is showing signs of foot drop. That is the most frustrating, its a catch 22.

Next to that on the frustration scale is the drug protocol. It is VERY detailed, with 3 hour eye exams 7 days before dosing, the day of hospital admittance and again on the day of discharge. There are also blood draws and temp checks every 30 mins for the first 24 hours after dosing, daily stool and urine samples and a bazillion ekg's (I think we counted 24 on the first day). That being said, we understand the need for all the documentation and my husband never complained. It also didn't hurt that mayo put us up in a huge suite with a lake view and a couch bed for me, our kids were up there with us every night and our son even stayed the night one night.

He goes back in for 2nd dosing on 7/11, and although he is dreading the extensive protocol, he is still going to do it. My fear is of him progressing more from laying still and not being able to move freely. The problem is that because we were on the neuro floor, their policy is that all beds have bed alarms, meaning he could not get out of bed to pee or stretch, without calling a nurse in there. We did cheat a little once we figured out that if I laid in the bed before he got up, the alarm would not go off But still. His walking and overall strength today vs the day before admission is very alarming to me. If anyone else is reading this and has any experience, please chime in. Could it be the progression is from the hospital stay or is this coincidence?

Thanks,
Kelly

 

[konagirl]

I would like to add....

I have not seen our Dr get excited about any of the 3 trials we were eligible for, this one included. Dr Boylan is a phenomenal Dr and I believe one of the best out there, but is very frank and certainly does not give off any false hope, which I appreciate. He told us on day one discussion about these 3 trials they were offering that he did not see any of them changing the face of medicine. We decided to go for it anyway, because really...a small chance is better than no chance, right?

I am saying this because, while in the hospital, he told us about the NP001 coming to a phase 2b trial this fall, and he seemed a little pumped about it. He told us the timing should be perfect for us, we will have ended this one just in time to allow for the 30 day waiting window to begin the new one coming out and said he wants us in it. I cant help but feel very optimistic....

 

[njk0407]

Hi Kelly,

I've been following your posts and am thankful for your update. ive had my eye on the upcoming NP001 trial so this definitely gives me hope to hear your Dr has some excitement about it. I actually read a book about a month ago called "Personal Trials" by Jeff Akst, which talked about NP001 and WF10 (I think is the name of it) so when our Dr mentioned it I recalled hearing about it. My husband was also just diagnosed, he's 36 and we have a 1 year and 4 year old. Pretty devastating as I'm sure you know. My heart goes out to your family. We are in the process of deciding on trials and my guts been telling me to go for the NP001. He's currently eligible for Tirasemtiv, but it would be for 1 year and that's a big long time risk to be getting a placebo so we are hesitant. This whole process sucks and I still can't believe it's happening. We need to make a decision soon though. I was also looking at high dose B12 injections and Lunasin and Actemra. I guess we are just kind of lost in all this. Good luck and God bless.

-Nikki

 

[njk0407]

Sorry the author of the book is "Jef" not Jeff. It's about how terminally ill patients took treatments into their own hands. They all had ALS, our Doctor, Dr Bedlack at Duke is also mentioned in the book so I thought that was pretty cool.

-Nikki

 

[phyllise]

Thanks for the update. I have not gotten another call from Mayo in regard to my admittance or denial. It is interesting that Dr Boylan is interested in the other trial? I pray it is the cure.
So glad your husband is doing ok through the trial but hate to hear of the hardship of laying still and muscle weakness. I understand totally.
Please keep me updated on progress I am going to try and contact them again next week to see if I can get a final answer.
Thinking about you and prayers for success!!!

 

[affected]

Kona I would have a good talk with your neuro before the next admission. If you are there with him during the day, see if you can advocate to keep him up and moving in some way while he is there.

Laying in bed plays havoc with the bowels too - if he is mobile, keeping up some mobility is really good for him as long as he isn't fatiguing himself.

When he is in bed you can also encourage him to do some bed exercises and stretching with ROM.

 

[konagirl]

Thanks Tillie, I will for sure be approaching the conversation again. Its not all on the hospital either though, they DO allow him to get up as often as he wants, they just require that one of their staff be with him. He is still very dependent at home, and works on his feet all day long, so he has a hard time with that. Plus, it is pretty awkward to have a nurse come in and stare at you just so you can stand up for a bit and stretch your legs, or if you just want to go look out the window. I told him though he has to get over that, he HAS to get up at least once every other hour this next go round and he promised he will. I am terrified of this next hospital stay and what it will bring.

 

[affected]

Kona can you negotiate with them that you will be with him once he is on his feet so you can supervise him like you would at home?

 

[konagirl]

I am definitely going to try again. Overall its a protocol for the entire floor, but some nurses were a little more lenient than others. They are not really used to have trial patients admitted like this, so there are some learning opportunities for sure. I wont stop asking though!

 

[konagirl]

Just in case anyone is following this CDG-0134 trial, I wanted to post a little update on where we are:

Chad was supposed to go back for his second dosing and second week in the hospital on 7/11. This has been pushed back to 7/25. The reason being is because Genetech wanted all 8 participants to complete their first dose, before anyone began their 2nd round. The 8th slot was just filled (at mayo with our Dr) so that person will be dosed soon in our original 7/11 spot, and then the round 2 doses will begin.

Something odd...There are only 8 slots for this trial. More may open down the road, but for now there are only 8. Our clinical coordinator shared some news with me this past Friday....she said this is not in writing yet, but there has been enough talk from Genetech on it that she feels comfortable sharing it with me: At the end of this trial, once all 8 patients have completed all 4 dosings, they plan to give all 8 of us the drug to take everyday, twice a day, at the strongest dose tolerated, for 4 weeks. Our clinical coordinator says she has never seen this happen, and even our Dr is pretty amazed.

 

[konagirl]

I don't know how to feel about this, or what to think......