Today we completed the Phase 2 trial running at Nijmegen with the intermittent regime. The trail continues since the last participant was on boarded in Aug 2011. Doc tells that results should be published by late this year. No other information available except for the few readings we took along the year.
We lost quiet some mts in the 6MWT over the year. The way my kid has slowed down; I am pretty sure that we were on placebo. Over that there were nothing but a few instances of bacteria and WBC in the urine samples.
However we start on the extension study in a month from now. The gap is needed to ensure that data for extension is isolated from the original study. A new baseline for the extension study, and we start with weekly shots. This time its for sure that we will gets the meds. NO MORE PLACEBO.
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This was exactly the point I tried to make earlier. After these GSK, AVI trials we will have 100+ boys in placebo groups (with measurements taken weekly, monthly etc. over a long period of time 1+ yr) hence very good historical data to be used in the next trials.
As far as I know the kids who stopped walking are 14 or 15. Not sure about this but got it as a comment from another parent.
My take away from the presentation is that kids who are showing improvement are showing it on a continuous basis at 93 week data check. The catch is looking at how the Prosensa presenter tried to draw a graph with his hand movement.
My guess is that variation is a lot tied to the age of the participatns
Yes, some sites are using general anesthesia for biopsies.
One would think that it would be standardized across the board, just like everything else.
Mindy said:
Yes, some sites are using general anesthesia for biopsies.
Nothing is standardized. Even the consent forms are totally different from site to site here in the US with one site promissing extension trial and another doesn't, for example. One site even has in the consent the fact that, if your son develops a condition due to the drug in the trial, any side effects that require treatment, GSK does NOT pay for treatment and most insurance companies in the US do not pay either. So you, as a parent, would need to pay by yourself. I doubt most of you have that in the consent forms you signed. So each parent needs to call his insurance company before the trial to find out if they would pay for treatment in case something happens to their son. Things do not seem standardized.
Andrea Cleary said:
One would think that it would be standardized across the board, just like everything else.
Mindy said:Yes, some sites are using general anesthesia for biopsies.
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