Apparently Santher and CHOP are excluding boys who use prednisone or deflazacort from their Idebennone trial.  Anyone know why?  It seems unethical.

 

https://www.duchenneconnect.org/index.php?option=com_content&vi...

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Paul, they are enrolling a number of steroid users and a lower number non. Enrollment starts with the non-steroid user group followed by steroid users. They noticed more improvement of pulmonary function in the non-steroid group hence the design.
This makes some sense, but these are children. Half of them are getting what is certain to be suboptimal care and, by defnition, they can't give informed consent. To justify making that decision for these people would require extraordinarily strong evidence. I recall that the prior study was pretty small. For example, how did they exclude the possibility that the variation in the increase in pulmonary function was the result of some confounding variable like mutations that were more or less associated with cardiac dysfunction?
I doubt anyone is going off steroids to participate in the study. There are plenty of boys out there not on steroids, and people have there reasons for this, ,...and I imagine it's never an easy decision.
Exactly. They enroll boys that are not on steroids at the moment. I am sure that they can find a good number in all those countries where they run the trial (they need to find a total of 50 (? don't exactly remember the number) or so from several countries). No one is asked to stop steroids to participate in this trial, they will enroll a steroid treated arm as well.



Jason Gautier said:
I doubt anyone is going off steroids to participate in the study. There are plenty of boys out there not on steroids, and people have there reasons for this, ,...and I imagine it's never an easy decision.
Paul, the statistical analysis should take care of the variation. I am sure that they collect a large amount of data (many variables) for each subject and then they run models. I trust these people know what they are doing, they should have statisticians on staff.

Don't forget that not all DMD boys are taking steroids at the moment. It might be difficult to enroll the non-steroid arm but not impossible since the trial is run in multiple countries. As everything else in DMD, using steroids is an individual decision... Looking at Mendell's minidistrophin trial for example, out of the 6 boys enrolled 2 were not on steroids...and that was a small trial enrolling only subjects from the Columbus area.



Paul Cliff said:
This makes some sense, but these are children. Half of them are getting what is certain to be suboptimal care and, by defnition, they can't give informed consent. To justify making that decision for these people would require extraordinarily strong evidence. I recall that the prior study was pretty small. For example, how did they exclude the possibility that the variation in the increase in pulmonary function was the result of some confounding variable like mutations that were more or less associated with cardiac dysfunction?
I'm not saying that it's never a good idea to refrain from steroids. There are any number of our boys who's families and doctors have determined that in their particular case, the benefits outweigh the risks.
This is the presentation I referred to. One can see the significant difference b/w the stroid and non-steroid group on slide 12.

They will enroll 40 non-steroid and 200 steroid treated patients.

http://www.actionduchenne.org/jsp/uploaded_files/documents/Conferen...
Thanks, Ofelia. So they intend, but have not yet opened for enrollement a much larger clinical trial some time next year where deflazacort and prednisone won't be exclusion criteria. My outrage is now doused, though it seems hard for me to believe that in a study with only 19 participants they could have convincingly filtered out confounding variables like the extent of heart damage before the test or the greater impact of certain mutations on the progress of damage to the heart muscle. In any event, the timing is such that no parent who already has their child on steroids would have any reason to take them off in order to participate in this trial, which was my main concern.

btw kudos for finding that in Santhera's website. I was unable to do so.
Have you noticed slide 10? Clinical trials with idebenone started 2005 and will end 2013... 7-8 years ... :(
Not to mention the 6 tablets daily dose...
I see that now. Since DMD progesses over years, there's probably no way to get convincing results in anything less than two years for what promises, at best, to be another weak tool like prednisone and deflazacort for slowing the progress by slowing the cell damage. One more bummer in a field of bummers.

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