Dexpramipexole and diaphragm pacer?

[jaws]

I've been on the Dexpramipexole study for about 4 months now. Based on my comparisons at PLM, I'm fairly certain that I'm on the placebo, but of course that's only a guess.

Because my breathing is labored at night, I've been prescribed with a BiPAP. I'm also in contact with clinic in Cleveland about having a diaphragm pacer installed.

Unfortunately, I've also just learned that if/when I have that procedure done, I will be kicked from the Dexpramipexole study. That outrages me, especially considering that the diaphragm pacer has been approved by the FDA for the treatment of ALS.

I cannot believe that the people in charge of that study can be so heartless as to force a choice like this on a patient. I am considering starting a letter writing campaign to plead with them to reconsider that decision.

Is there anyone else on the Dexpramipexole study also facing a similar choice?

Thanks,
Aaron Winborn

 

[trfogey]

The dexpramipexole trial was not designed to account for the diaphragm pacer. Leaving your data in the pool after you get the implant would violate the approved rules for the trial. It's not about being kind or cruel. It's about getting accurate data so that we know whether the drug works or not.

You've discovered the downside of participating in trials. You sign up for a trial and you give up some of your freedom of action. You're not the first one who's faced that choice and you won't be the last. Sorry it's worked out this way for you. But please realize that there are a lot of folks out here that don't qualify for either program and would gladly trade places with you to make that choice.

 

[Alyoop]

They are not being unfair at all. One of the primary or secondary endophyte for the study may well be respiratory function. If you use a device to alter the course of the illness then they can't know if it's the drug helping or the device. It's nothing to do with the device being FDA approved.

I know it seems unfair, but that is the reason you have a very long consent to sign. The data would be meaningless and the effect or lack of effect, of the new compound would not be clear if you and other people with diaphragm pacing devices remained on the study

As the drug is yet to prove it's effectiveness, it would not generally be provided compassionately by the company, after you have been withdrawn.

I have worked extensively with Biogen and they are very good at continuing supply of a drug, that has positive effects, at the end of the study.

I have just had a young patient who had to come off his study to start a family. The drug he is taken, has just had the analysis done and it is the most effective drug for ms so far ( not on market yet). It was devastating for me that he had to stop the drug when he has been so well for 4 years, but it was the choice he decided to make.

I am sorry that you are facing such a difficult dilemma, but a letter to Biogen will not help. They have no choice.

 

[sadiemae]

Jaws, be thankful for that you at least get a chance at these options. I started researching the Pacer over 2 yrs ago, but approval came to late for my husband. Same with any trials out there, he has had ALS too long to be a candidate for any of them. All we can do is the renegade oral sodium chlorite experiment. I would do the Pacer if it was me. It will keep you going long enough to get the Dex when and if it is approved.

 

[Alyoop]

Sorry my post above has suffered from my iPad endophyte ( whatever that is) was supposed to be end point. Sorry

 

[jaws]

One might say the same thing about using a BiPAP, which is another FDA approved device used to improve respiratory function in PALS. But what they do with that, is adjust the data taking that into account. They would be doing the same thing with diaphragm pacer.

Even, if I play devils advocate, and make a claim that there's not enough data for the pacer at this point, I am certain there will be plenty of data by the end of the study. For instance, at my clinic in boondocks Hershey, PA, there are currently 3 patients who have already had the procedure done, and in the 2 months since its approval, there have been 3 others besides myself who have begun the process to have the procedure done.

To be conservative, we might guess that that translates into, say 150 PALS with the pacer. Extrapolating that outward, even if we assume from the doubling a linear progression rather than exponential, there should probably be upwards of 1000 or more people with the pacer by the end of the study, or at least by the time they are processing the results.

Thanks,
Aaron

 

[trfogey]

Jaws, be thankful for that you at least get a chance at these options.

"Grateful" doesn't seem to be a word in jaws' vocabulary, sadiemae. Neither does "compliance" or "contract". From the looks of things, those are words that people other than jaws should live by.

Sorry, jaws. Not feeling much outrage over your situation, nor much sympathy for your cause. DPS approval was in the FDA pipeline when you signed up for the trial you're in. Maybe you should have stayed out of the trial and waited for DPS to come online.

 

[sadiemae]

BiPap use kept a lot of folks out of the NP01 trial. The Pacer will be helpful to about 3000 PALS.

 

[csgam]

I have also been in the Dex trial for four months and am able to get the Pacer implanted as early as next month if I am willing to go off the trial. Theory is Dex can extend life by 39% if proven correct but 50% chance I will be on the Placebo for up to 14 more months before switching to open label usage and being 100% sure I am on the drug.

By getting the implant my quality of life is improved by extending the time before a trache is required. Trade off is the 18 month Dex trial has to be completed and roughly another year will pass after before the drug comes to market IF the Phase 3 study proves the theory is true and approvals are ok'd that's over two years from now.

Even though though the Pacer is considered experimental for humane use in ALS patients, we know it works. I am strongly leaning toward the Pacer. If you really wanted to stay on Dex for now you could have the surgery and just not hook up the box to the wires and not operate it until later. May defeat the purpose but just a thought.

 

[retiredmus2010]

Jaws: May I ask what clinic in Cleveland?

 

[trfogey]

Jaws: May I ask what clinic in Cleveland?

Dr. Onders is affiliated with University Hospitals in Cleveland, more specifically Case Medical Center, according to most articles I've seen about the diaphragm pacer.