Trial protocol

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Hal

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Question …. Is anyone else concerned about the timeline protocol that exists with most trials? While I am one year into a diagnosis, I am now hitting the 3 year mark in regards to when I perceived my initial symptoms started. This means that I essentially will no longer qualify for any trials. Given ALS effects everyone in a different way, it is hard to understand the science behind creating a timeline “cut” especially when pretty much all of the trials are not able to recruit enough patients to generate a data stable sample size. Any thoughts?
 

Nikki J

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The issue is progression rate with PALS who would otherwise qualify for function level. If you are three years in and not approaching end stage then you probably aren’t going to change frs score fast enough to give a clear signal. Delay to diagnosis is also correlated with slow progression which is part of the reason they use symptom onset most of the time. We desperately need a decent biomarker. If we had one this wouldn’t be an issue. I don’t see diabetes and hypertension trials with diagnosis onset restrictions because they have easily measurable parameters Do I hate this? Absolutely. All the more because I really believe things would work better on less aggressive disease. It only makes sense. Anyone who can and has an opportunity do biomarker trials. We are getting closer to validating some body fluid and imaging biomarkers which would lead to change
 

Hal

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PALS
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I understand … but given the limited trial sizes, it makes significant more sense to create “A” level and “B” level trials that are conducted at the same time. By blocking those that have a slow progression version of ALS, we may also be denying those that may benefit the most from new treatments.
 

Nikki J

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That doesn’t address the issue of slow progression not generating a strong signal. you would have to make the trials very big and very long to see anythng which isn’t financially feasible. They do collect data on expanded access which gives information about non trial populations. As I said I don’t like it. I never got to be in a trial and I’m far too out to ever qualify now
 
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