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One year on Phase 2 of GSX/Prosensa Trial

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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Permalink Reply by Ofelia Marin on February 9, 2012 at 9:23am

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.

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Permalink Reply by Ofelia Marin on February 12, 2012 at 10:54am

CureDuchenne had a scientific summit yesterday and I was able to watch part of it online.

This is what they said about the 12 boys in the first GSK trial, now in open label extension at week 120:

Out of 12 who started 10 are still in extension at week 120. He did not say why the other 2 are out.

4 out of 10 continue to show improvement in 6-mwt.

1 out of 10 is stable.

3 out of 10 declined.

2 out of 10 were not able to complete the test.

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Permalink Reply by Tulika on February 12, 2012 at 11:25am

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

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Permalink Reply by Mindy on February 16, 2012 at 4:47pm

Yes, some sites are using general anesthesia for biopsies.

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Permalink Reply by Andrea Cleary on February 16, 2012 at 8:19pm

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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Permalink Reply by Ofelia Marin on February 17, 2012 at 4:32pm

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.