Tirasemtiv trial

[Vikman]

So we met with the ALS clinical trials coordinator at the hospital for special care in CT. They will be enrolling patients in the phase III Tirasemtiv trial and actually will be enrolling the most patients of any sites worldwide. They were also one of the sites for the phase II trial of Tirasemtiv as well.

Anyway, the doctor was saying the drug seems to show a lot of promise, she had patients during the phase II trial that showed improvements while on the drug. For instance, one of her patients was unable to lift her arm to brush her teeth but after being on the drug she was able to. They are just waiting for IRB approval and will start enrolling within the next few weeks. Think I'm going to get my dad into it.

 

[notBrad]

I go for my screening on 12/1

 

[scaredwifetx]

Steve has been in the trial for a few weeks. He received the Tirasemtiv for two weeks and is now in the double blind study so we do not know if he is actually getting it, a lowered dose or mx dose. He did not have an side effects the first two weeks so it is hard to know for sure. The trail is to last a year. Haven't noticed any changes so far but will keep everyone updated if we see any changes.

 

[hjlindley]

Just be aware that that drug lowers your blood pressure and enter reacts with tizanidine if any of you are taking it.

 

[scaredwifetx]

That may be why Steve's blood pressure was lower at clinic. With it being a double blind study we are not sure if he is now getting the drug but for the first 3 weeks we know he did. He has not had any side effects that we know of.

 

[scaredwifetx]

Steve's FTC test went from 90% at clinic in August to 77% last Friday. That worries me some and I thought this drug was in trial to improve breathing but not sure. I have not had the time to do a lot of research on this trial.

 

[Vikman]

Steve's FTC test went from 90% at clinic in August to 77% last Friday. That worries me some and I thought this drug was in trial to improve breathing but not sure. I have not had the time to do a lot of research on this trial.

How long has he been on Tirasemtiv? Maybe it needs a little time to stabilize FTC or improve it(if it works).

 

[scaredwifetx]

He has been on the Tirasemtiv or placebo for about a month. Today he had his first episode of trouble swallowing food. It was corn. not something tough but small I guess.

 

[lgelb]

Corn, rice and/or peas are often the first foods that need to be off the menu. If he likes corn, try moist cornbread.

 

[scaredwifetx]

Thanks Laurie. Before yesterday he did not display any swallowing issues. He has been very irritated, anti-social and walking abilities have declined over the past few days. His dosage for the Tirasemtiv trial doubled earlier this week. We don't know what dosage he is really getting or if it is just placebo so it makes it hard to guess what is going on. My mom said yesterday to me that Steve's personality is changing and his brother is now also noticing things like the walking and mood. I am not sure what to think or do. If he was not in a trial we would know his progression was the reason for the sudden changes. Of course, he does not want to talk about any of it and gets quite irritated with me when I mention anything. I noticed his lips kind of twitching and asked him to stick out his tongue. He did but when I seen the twitching he immediately got mad and told me that he did not want to know anything and to leave him alone about it. Last night he blamed me for turning in bed and keeping him from sleeping. I am thinking it is time for us to move into our twin beds.

I am not ready for all of this and wonder if the changes this week are due to the trial drug or if we are now on the fast and crazy ride.

 

[bear1973]

Sorry to hear about Steve. It certainly is confusing given that you don't know if he's actually on the drug. Can you ask the researchers/your neuro if the drug could be causing these effects...? if that is the case, maybe he could consider withdrawing from the trial.

I have been offered to be in the trial and am just waiting to hear back if eligible based on the supplements I take....
Sad thing is: too bad it takes 3 YEARS before the final results are known. Jeeezzz......we don't have that long!

 

[affected]

I know it's really hard when things move so fast, you are just kind of running and never feel like you catch up for more than a few days. The denial and withdrawal by Steve then make it extra hard.
Are you saying you don't know if the swallowing problems could be a side effect?

 

[lgelb]

Accelerated progression is always a risk in trials (minocycline being one example), but was not seen in the earlier tirasemtiv trials, obviously. Once someone is on anything, from supplements to rx, you have no time machine to know whether the course of the dz has been changed, better or worse than it would have been otherwise. All you can do is deal with what comes and try to stay with what seems to work best.

Remember, this rx did not meet its primary endpoint in the last trial and was only moved forward because of subgroup/secondary endpoint efficacy. That suggests heterogeneous effects, not the same effects in all PALS.

As for swallowing, walking difficulty and twitching, as with all the other ALS neuromuscular symptoms it is very possible that something is no issue one day and is the next, so I would not necessarily put it down to the drug, though the more of these that occur exactly at the same time the more it's tempting to identify the new agent (esp. if it's the only new thing for some time) as the culprit. Of course, it's always his option to continue on it or not.

Certainly discuss what started when with the investigator (type it out chronologically w/ dates so it can be entered into the trial database accurately as well).

 

[notBrad]

Contact the trial and see what they have to say.

 

[scaredwifetx]

Thanks everyone. I am going to his next trial visit and will mention it all to them. I am guessing this has nothing to do with the trial and is just weird timing. Thanks for keeping me one nut away from going crazy one again. I am trying so hard to stay strong and stay focused.