Efgartigimod (Vyvgart) - what could the trial data possibly tell us?

If this was mainly for POTS only some patients presumably happened to have PEM. Unless they screened for that specifically, I don't think the data is very interesting.

ChatGPT says the drug reduces IgG levels which is interesting though.
 
EndME said:
Since it seems that just as many people in the placebo group got better you currently probably have to assume that roughly half of the people that are reporting their improvements not based on the open label part actually didn't receive the drug and the ones reporting improvement only based on the open label part of course needn't say anything.
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Only a third of the participants didn't receive the drug during the blinded trial. After the blinded trial, the participants were given open label access. I'm not sure if people were told what arm they had been in then or not.

Four of the ten people mentioned in the article reported that they only started getting better during the open label part of the trial. They may well have been part of the third who got the placebo in the blinded part of the trial. But they may also have been part of the two thirds who got the drug in the blinded part of the trial.
 
Hutan said:
Only a third of the participants didn't receive the drug during the blinded trial. After the blinded trial, the participants were given open label access. I'm not sure if people were told what arm they had been in then or not.

Four of the ten people mentioned in the article reported that they only started getting better during the open label part of the trial. They may well have been part of the third who got the placebo in the unblinded part of the trial.
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Indeed, I just re-noticed the one third vs one-half split. From what I've heard nobody was unblinded. Sure or they all got the drug previously already, the chances of that are higher, one day we'll know.
 


Fwiw a reddit comment about the study questionnaires from a participant.


I'm not saying this drug definitely worked but I think there are a lot of elements of this trial fiasco that fit with what we have talked about in terms of problematic trial design.

I think the NIH would do better to fund a new, better designed phase 2 of this than say, yet another viral persistance study.
 
EndME said:
Get access to the data and analyse it, but don't already demand redoing trials on the basis of your own beliefs.
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If they truly believe the drug helped them, and the drug company are refusing to release the data, what else can they do but call for another trial?
 
V.R.T. said:
If they truly believe the drug helped them, and the drug company are refusing to release the data, what else can they do but call for another trial?
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From what I know the drug company cannot refuse to release the data. I think there's even a fairly short limit on the time duration for how long data can be withheld. They are forced to relase it by US laws, the problem is getting timely compliance. I'm sure there are probably many gray zones and that it will be a lengthy and unrewarding process that shouldn't even exist in the first place because the data should just be made available, but I think getting compliance will be probably still be a lot easier, quicker and cheaper than readoing the trial. Argenx has not released the data because it is negative, not because of other reasons.

Other than that I wouldn't be surprised if someone like Federowski has actually seen the data. So there possibly are already people in the know.
 
Is there any reasonable chance that they would respond to a request by e.g. @Jonathan Edwards to view the data without being allowed to share it? At least we would know if it’s worth pursuing rapid publication.
 
Utsikt said:
Is there any reasonable chance that they would respond to a request by e.g. @Jonathan Edwards to view the data without being allowed to share it? At least we would know if it’s worth pursuing rapid publication.
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Maybe, but I don't see why. Why would they care? I could obviously be wrong but my guess is the following: The trial didn't only show that the placebo and treatment were equal off, but that placebo even did better than treatment. As Hutan has mentioned companies tend to torture data even when it is negative and make and press release sound impressive. The press release here has been extremely short.
 
If the drug had a significantly useful effect I think it very unlikely that questionnaires would fail to pick up a signal even if they were less than ideal.
 
Wasn't the trial terminated in the middle of the open label phase? I'm no expert but you tend to only do that when you are convinced of there not being an efficacy precisely because most costs have already occured.

I can understand people talking about problematic trial design and that is justified if the arguments are reasonable, but none of the points that are brought up in the article are well founded. It's just the repetition of the same nonsense we see again and again. Afterall the most problematic part is that it is a "Long-Covid POTS study" which is what the article leaves completely unaddressed.
 
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