NP-001 Promising Phase 2 Clinical Trial

[Trixie]

Hi Everyone,

I am writing to see if anyone else is aware of this new very promising clinical trial for ALS. I believe this could be the drug we have been waiting for all these years. Please contact your clinic or doctor to see if they are recruiting now for this trial. I would love to be in this trial but am excluded because of 7 year symptom onset.

What can we do as ALS patients to bring this drug to market is quickly as possible. One thing ee can do is volunteer for the trial and another is to contact the CEO of neutraltus.

Here are some of the points you might include in your e-mail. The CEO, e-mail address is at the end of this post.

1) Pursuing a drug for ALS, even though the market is much smaller than for many ailments.

2) For the atypical speed with which they completed phase I and progressed to phase II for NP001.

3) For expanding their inclusion criteria to 36 months post-onset (was only 24 months).

And, to:

4) Please have their Data Safety Monitoring Board (DSMB) frequently and closely monitor the results to detect the apparent effectiveness of NP001 and consider changing to open label as quickly as possible, as it may be unethical to continue placebo for 1/3, while many seem to be improving on the drug (and not just experiencing slowed disease progression). The ALSFRS-R scale is crude and may not enable indications of subtle, but real, improvements which do not result in a whole point increase in the scale, but will over a longer period result in possible gains or slowed/no loss.

5) Please consider extended open label use for the volunteers of the study at a minimum, and consider compassionate use to anyone disqualified from the likely future phase III.

6) Express that the ALS community is very excited about about the few reports of effectiveness through either forums or other communication. It may help Neuraltus Pharmaceuticals secure any necessary additional investment funding, if necessary and essential to continue their momentum towards phase III and FDA approval.

Direct Email for Andrew Gengos [email protected]

. Thanks for being so patient with this new first long post.

Trixie.

 

[sadiemae]

We did the phase 1, but our onset date is over 2 yrs, so no Phase 2 for us.

 

[Trixie]

Dear Sademae,

Did you notice any changes while in phase 1 NP001 trial? Perhaps you could write to the CEO and request open label use of the drug?

The more people that write to Neutraltus about open label use and compassionate use for all those not eligible for the trial, the better our chances will be of succeeding.

I wish everyone would just take a few moments out of your day and write a thank you to Neutraltus for bringing this drug so quickly into phase 2 trial and hopefully into phase 3 quickly for FDA approval.

The best advocate for us is us.

Trixie

 

[sadiemae]

Nothing at all, however they said he would need at least 20 doses to notice anything. Phase 1 was only 1 dose.

 

[george123]

Sadiemae, I'm in phase ll. Last week during infusions our trial coordinator said they recently had a teleconference with Neuraltus and the 24 month from symptom onset enrollment criteria was changed to 36 months. All other requirements including 70% or greater FVC remain. They made this change so they could complete enrollment of the remaining available slots, as of last week they had 55 people screened or enrolled out of 109 total they are hoping to enroll.

There is a great deal of enthusiasm on various ALS forums about this trial!

 

[Karigav]

My dad is in the phase 2 study. I am ditting here with him now as it has made him very ill. We are.not sure if it is drug related or that the als has all of a sudden progressed to this stage. He went from walking and living somewhat normal(other than needing assistance in the shower and getting dressed and holding things) to bedridden and helpless. He is having a lot of pain and they have given him the phentenol patch and oral meds, but nothing is easing the pain. I dont think he is going to make it for another treatment. He has had 8 doses so far.

 

[sadiemae]

Unfortunately the 70 FVC is no longer in our vocabulary.

 

[rknt50a]

Just in case anyone is considering a trial, here is a quote from a patient who is currently enrolled in the NP-001 trial (in reaction to some positive indications from some participants):

Recent word is that there were about 50 enrolled. So, of those we can expect about 16 to 17 in each of the three trial arms (placebo, low dose and high dose).

Spread the word. Statistical significance requires sufficient participants. We need to fill enrollment ASAP. A reminder that the enrollment criteria allows onset up to 36 months and Neuratus and/or study sites may pay for hotel and travel expenses for those not close to a site.

Here is the blurb on clinicaltrials.gov

Please keep in mind that the current criterion includes patients up to 36 months from onset.

 

[Happyphysicist]

My dad is in the phase 2 study. I am ditting here with him now as it has made him very ill. We are.not sure if it is drug related or that the als has all of a sudden progressed to this stage. He went from walking and living somewhat normal(other than needing assistance in the shower and getting dressed and holding things) to bedridden and helpless. He is having a lot of pain and they have given him the phentenol patch and oral meds, but nothing is easing the pain. I dont think he is going to make it for another treatment. He has had 8 doses so far.

Are you at the University of KY site? I am there too. Can you tell me if he had any symptoms a few hours after the infusions?

I would assume that if the neurologist at that site thought it had anything to do with the drug he would have stopped the trial for him right away.

Can you tell us more? It will be helpful in case other participants have a similar experience.

 

[sadiemae]

Only thing Les felt was a weird dizziness. Also that night he had real bad night sweats.

 

[joni51]

These trials are all too far for us to travel

 

[Bad Balance]

I am not familiar with the anticipated results for this drug. Is it simply a 3-4 month extension of life or something more significant?

Thanks.

- Jerry

 

[Karigav]

Its been a long week. I appologize for the delay. We will know more after tommorow (hopefully) but they are not sure if this is drug related or not. Take confidence that the neurologist heading up the study is very aware of dads condition, and is following us very closley to see if there is any possibility that the drug started all the issues. It may be a freak coincidence that the drugs came right before the sickness did. I love the neurologist that is in charge of the study and whole heartedly believe that he,would stop if he thought that the drug did this. So for now, we sit and wait. I wish we could see him regularly, but he is not in our jurisdiction with the VA. And yes, he is at uk. They are awesome there!

 

[Happyphysicist]

I am not familiar with the anticipated results for this drug. Is it simply a 3-4 month extension of life or something more significant?

Thanks.

- Jerry

The drug seems to stop the progression of the disease cold. The longest anyone has been on it was 6 months. During those 6 months the FRS scores for those patients remained the same. There are 3 people on PLM whose FRS scores have gone UP a little since taking the drug (of course they might be on the placebo, but its unlikely).

If you are eligible for the study please enroll!
HP

 

[Happyphysicist]

Its been a long week. I appologize for the delay. We will know more after tommorow (hopefully) but they are not sure if this is drug related or not. Take confidence that the neurologist heading up the study is very aware of dads condition, and is following us very closley to see if there is any possibility that the drug started all the issues. It may be a freak coincidence that the drugs came right before the sickness did. I love the neurologist that is in charge of the study and whole heartedly believe that he,would stop if he thought that the drug did this. So for now, we sit and wait. I wish we could see him regularly, but he is not in our jurisdiction with the VA. And yes, he is at uk. They are awesome there!

I have to say that the ALS clinicians at UKY have to be the best I have met. Dr. Karsarskus is one of those Dr's you trust with your life. (I might have misspelled his name here). The rest of the team are equally thoughtful and caring. Your father is in good hands.