Clinical Trial Therapies

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Nikki J

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Both. There are more trials now than before which is good in many ways. Trials often also have exclusions for symptom duration which is a problem if you took time to diagnose. And feeding tubes, bipap and compromised breathing. And on the other side the Nurown trial when I was diagnosed had an exclusion for people who had been symptomatic less than a year. but mostly they want people who are relatively early in disease and they don’t want anyone who is slow in progression
 

GXTrex

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I see. Did anyone on here get into a trial?
 

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I got into a trial not long after I was diagnosed, but most (drug) trials I've looked at since want you to be fewer than 2 yrs from diagnosis, so I'm ipso facto excluded.

Ed
 

GXTrex

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Ed,

Did you continue the trial meds afterwards? Did they ever tell you if you received the drug or a placebo?
 

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No--I had bad side effects from an open label pretrial so had to drop out. The company as I recall had to withdraw the drug (tirasemptiv or something similar)
 

Ed340hp

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I was in the Vitality ALS (tirasemptive) trial that was held back in 2016-2017, the first ALS trial in years (before the Ice Bucket funds kicked off interest in potential treatments). It was a 48 week double blind trial, and a 48 week open label extension that was shutdown half way through the year after the endpoints were deemed unsuccessful. The trial started with almost 750 participants across 17 countries, and ended with about 350 participants when they shut down the open label extension trial. I had to return the unused drugs when they ended the trial. I have asked for, and have not received, any of my quantified trial data (monthly ALSFRS, FVC, SVC, strength tests, and blood tests), and the trial data was maintained separate from my ALS Clinic chart records.

I still have no confirmation that I was, or was not, in the placebo group. When I titrated up to the maximum tested dose in the open label extension I noticed some of the side effects (jitters), but the blind trial tested three dosage levels and the placebo, so I could have been a lower dose participant in the trial. The treatment goal was to stimulate fast twitch muscle to preserve function, not to preserve neurons. I was told the efficacy endpoints looked good, but only at the maximum dose and the side effects at that dosage were deemed too severe for the benefit.

I was told I appeared to be a slow progression PALS three months after the trials ended. During the trial I was told to stop my prior exercise habits to avoid tainting the results, a lack of movement that I believe accelerated my progression in small muscle groups that may have been preserved better with ROM and light effort exercise (what I slowly returned to with great difficulty, after the trial, with some restoration of function).

I advise anyone in a trial to document a personal log of any test results that can be copied down as the testing is performed. The official test data is the property of the trial sponsor (drug company), and you do not have the rights to official copies of the trial data collected. If you want to document your progression or improvement, you have to do it yourself.
 

GXTrex

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Ed,

I am very surprised they would not provide any of your own data from the trial, and even if you were in the placebo or not. Did they provide the information (ALSFRS/FVC/etc) at the time it was quantified and just expected you to keep track of everything yourself?

If given the option theoretically, would you partake in another trial?
 

richdees23

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can anyone share info regarding the healey platform trial? i was told a year ago i was a great candidate, yet i have not been enrolled.
i understand covid has played a large part in its delay, but everytime i inquire about it, i get the "be patient..."
i don't mean to vent (different forum, i know), but i'm just looking for some concrete, valid updates.
thanks!
 

Nikki J

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Are you on a waiting list? What site are you waiting for? There are weekly webinars about the trial and there are 2 people who you can contact if your site is not answering you I sent you a link
 

Ed340hp

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They did not provide the information (ALSFRS/FVC/etc) at the time it was quantified. I expected them to share my data with me, and even had a verbal commitment from the Trial Manager at my ALS Clinic to provide me the data after the blind trial ended, but my data was restricted. Since then I learned to keep track of everything myself.
 

EricInLA

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Ed340hp - It is very distressing that the trial sponsor would treat you that way, considering you are giving so much of yourself for the benefit of their drug research. I'm getting the sense that I really need to advocate for, and look out for, myself as I (hopefully) enter into a trial soon. I also suspect that exercise is helping to slow progression for me so I will continue my regimen.

RichDees23 - I am in the same boat as you. Just waiting for the Platform trial to get moving at my local hospital. I bug them every once in a while, and I get the same response - "yes, we've got you right here on the list, and we will let you know as soon as we start the trial." Meanwhile I've got a ticking clock here because I'm 4 months away from my 3-year symptom anniversary. It's frustrating but I don't know what I can do. Covid has messed with everything. I even contacted Platform Trial 'headquarters' in Mass. but they just referred me back to my hospital. I have my 6-month multidisciplinary clinic coming up so I will further inquire then.
 
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