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notBrad

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OK, I go on Tuesday 12/1 for my initial screening for the Tirasemtiv Phase III trial but I check my email today and received this for the NP001 Phase II at Cutter.

<snip>

Dear Brad,

Thanks for your interest in the NP001 study and your patience as we get the study organized.

I am very excited to tell you about this trial.

The purpose of this drug study is to see if the drug NP001 can slow the progression of weakness in ALS. The hope is that NP001 will keep ALS patients stronger for a longer period of time. It is a clinical trial, an experiment, so we don’t know the answer until the trial is completed.

This study will be conducted at about 8 sites across the country. There will be 60 patients total. Each site will have 6 to 10 patients. We expect that most sites will begin enrollment in January. I believe that the site closest to you will be at California Pacific Medical Center in San Francisco.

NP001 is given through a vein in the arm. There is a screening visit to see if you qualify and then you would come back to the clinic within 28 days to start the infusions. Each infusion takes one hour and then you have to stay at the clinic for 2 hours after you finish the infusion. The infusions are given in cycles on consecutive days. You go home each night – this is an outpatient study. The first cycle is 5 days in a row. A month later you come back for 3 days in a row, and then monthly for 3 days in a row for a total of 6 monthly cycles. That would be a total of 20 IV infusions. Finally, you come back a month later to get checked out and exit from the study.

There are criteria that you must meet to get into the study. Some of the main criteria are:

· Diagnosis of ALS.

· ALS weakness started less than 3 years ago at the time you enter the study.

· Good breathing, FVC above 65%.

· Have good veins.

· Not using a machine like BiPAP or CPAP to help you breath at night.

· Not have a feeding tube.

· Not have any other significant illness.

So you can see that this study will be very time consuming and may not be a good fit for everyone. I hope you remain interested in this study and I am happy to answer your questions.

</snip>

Thoughts, advice, etc... ?

Thanks,
Brad
 

BlueandGold

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Precisely why I opted out Brad. I would have had to go to Duke, which is a 6 hour trek for me.also, time for my feeding tube so not for me. Good luck with your decision.

Vince
 

Green Queen

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Sounds full on Brad.
What does Vicky say?
All the best with the decision making.
God bless, love Janelle x
 

Nikki J

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Tough to decide. I am thinking all being equal you wanted to try NP001? But it sounds burdensome physically and financially due to the travel. And No guarantee you would get into the trial or that it will really start enrolling in January. Good luck in choosing
 

notBrad

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Thanks all!

Vicky is leaning towards the NP001. Travel is not an issue as we bought the motorhome in anticipation of attending clinics so which ever one we choose we just head there and settle into a nice RV park.

I think I'll just ask some direct questions of both researchers and my doctor and take it from there.

Will let you all know.

Take care,
Brad
 

mich5

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you might want to do a search on this forum using np001. there were members on this site who were in phase 1. i remember a side effect was dizziness. members here had extreme dizziness, one could not walk from the dizziness (he actually had a fall). also search using sodium chloride, i think there was some discussion using that term. i think pearshoot was in phase 1 and he posted that he increased his hand strength during the trial but lost it all within a few weeks of being off the drug - but dont quote me on that unless you find it. :) dont recall anyone being on the tirasemtiv . . . alsuntangled has a report completed on np001.
 

lgelb

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Have you had your interleukin-18 and lipopolysaccharide (LPS) levels drawn yet? It looks like those are the major markers for possible response in the trials to date.

In the forthcoming Phase II trial (delayed for several months now), the primary endpoint is not clinical efficacy but effect on those biomarkers specifically.
 

notBrad

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Have you had your interleukin-18 and lipopolysaccharide (LPS) levels drawn yet? It looks like those are the major markers for possible response in the trials to date.

In the forthcoming Phase II trial (delayed for several months now), the primary endpoint is not clinical efficacy but effect on those biomarkers specifically.

Would you think there'd be sufficient dosage differences to affect whatever benefit(s) I might see?
 

lgelb

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No, it should be the dosage(s) they will use in future trials (part of the reason for testing biomarker effects is to further justify their use as surrogate endpoints in future trials). I just wanted to point out that this is not primarily an efficacy trial, in case people were wondering why it's so small. But it will either support or cast grave doubt on the wisdom of continuing development.
 

notBrad

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rknt50a

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At the ALS MND symposium a year ago when McGrath presented data from the old Ph II trial, he showed that participants with high IL-18 and LPS were the responders. A question from the audience asked whether they would limit the next trial to high IL-18, LPS people and McGrath indicated they would. That wasn't official trial design, but...

From a bunch of participants in the first NP001 Ph II trial, they reverse-engineered data at plm and also figured out that some "high side effects" people (primarily based on stinging at the infusion site) were getting positive responses. They were pretty sure that those were the high dose group.

In that trial there was a washout period after the treatment period so that they could watch the biomarkers. Several people I knew who had done well in the trial (with actual improvements in some symptoms) crashed terribly during the washout period without NP001, but Neuraltus was able to watch the biomarker while they crashed.

A crueler trial design I cannot imagine.

And product was never again made available to trial participants.

Perrin made a comment at the ALS TDI summit recently that he does not know what Neuraltus will be able to prove with yet another small Ph II trial.

Some misc info, thoughts fwtw.
 

lgelb

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Rknt,
Needlessly cruel trial design is seldom approved by the FDA, IRBs or the patients signing on.

This compound is hanging on by its fingernails, as is the company. Is it so surprising they would feel a need to validate the mechanism by which the drug might actually work? To stratify PALS so people who won't benefit won't waste their own very limited time and everyone else's resources?

Even in ALS, if they didn't do these things, it would have no chance of marketing approval or reimbursement once the biomarker relationship became known from the previous trial. Are you saying they should have faked the data?

Re small N, there's always a tradeoff between speed and size. In pharma, we try to kill [development] quickly if we must kill at all, because it's less cruel all around. But, beyond that, data delayed too long can kill a cash-burning drug all by itself, and since the ALSA is reportedly supplying half the trial budget...and what has Steve Perrin brought to market?

As for not doing an extension, do you think the FDA would have approved one, given significant biological differences between responders and non-responders, in Phase II? Who do you think would have funded it? And...ummm...they're still dose-finding.

It would be nice if this were a Phase III trial, powered and viable to be that, but you can't do Phase III without approval. And you don't want to scrape pennies together even to design for Phase III unless you're certain that you can ultimately gain regulatory approval with that program. I can see clear reasons why Neuraltus would not be, at this stage.

In short, they're doing the trial they can get funded, that seems to advance the program, with a variety of constituencies. It's real life. If they can't sell/out-license the thing (which is more likely if the mechanism is confirmable), it's probably going to wither.

People at PLM can deconstruct all they want, but by helping each other unblind dosing arms, they're only compromising data quality, and who are they helping? They might funnel that energy into raising some R&D $ IMHO. And those are my misc. thoughts tonight.
 

notBrad

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Ultimately I have decided on the Tirasemtiv because a) it's Phase III and will last over a year and b) the NP001 people are very flaky about getting back to me and the trial actually has no hard date for starting (all they'll say is that "it's slated to start enrollment sometime in January").

Thanks all!
 

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Hope tirasemtiv does you some good, if you're randomized to a tx arm. As you've probably seen, Deb's husband Steve is in the trial, and Bear is being evaluated.

--Laurie
 
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I did not reply here because I just didn't know anything much at all about either trial.
But I want to say I'm so happy you feel you know which one you want to pursue now and I hope so much that we get good news out of this trial and especially that you personally get the drug and the benefit.
 
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