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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Thanks so much for sharing Tulika.

Must you wait along with the rest of us for the final results before you know if your child was on placebo arm?

-David

Thanks for the update. We have 17 visits left. We go to Lille, France (live in UK, but from US). Fairly positive he's on the drug, but haven't seen any great results.

Thanks Tulika! I hope you see something positive in the extension.

David, Till the results are made public, we only get to see the data gathered at the site of our child. Also since every one is supposed to be blinded from data the docs and nurses dont look back at the past data. We did not ask for the full file of data data since we have the basic readings of 6MWT and other physical tests.

Officially no one knows whether we were on the placebo or the drug, but everyone's guess is that at our site the placebo control was our son. We also have to wait for the results to be made public to get a confirmed answer on whether we were on a drug or placebo.

Tulika, all boys on the drug had reaction at injection site every week and protein in urine, as reported by Prosensa/GSK previously. So if you son did not have any, you can be 100% sure he was on placebo. This trial although blinded is so weird b/c everyone knows who is on placebo due to lack of reaction after injections. I am looking forward to the extension, your son has the same mutation (deletion of 50) as mine and Laurie’s. Praying that you can see something positive while on the drug!

Well, every boy in that trial knows that he is on placebo or the drug, hence I do not see how whatever is posted here can change that. The drug's side effects are published and presented to all conference, hence no surprise to anyone.

Tonya Carlone said:

I fear with all this talk about the trial and whether or not a child is on the placebo arm or on the drug that it could comprimise the trial.  The fact is we need the trial for all of our boys not just for exon skipping 51, but for the future exons to help other boys. 

Tulika - how old is your son? I am very sorry that he has declined over the past year.

 

The Duchenne community owes you a huge debt of gratitude.

I don't quite understand why they need the month off? Can't they just use the completion data from the phase II as your baseline for the extension? Can't one decision be made on behalf of these poor boys on placebo rather than what the drug company wants? They got their 48 weeks of (most likely) placebo, and somewhat less than pound of flesh (literally) - just put the boy on the drug already.

Well, all I can say is that the folks involved in the process are equally commited. As parents we have the luxury to vent out out frustrations. This trial has made me realize that almost everyone in the drug discovery process shares the same passion. However there is no other way but to be extra careful. I have seen the kind of effort folks at the hospital, Prosensa and GSK have put in when my kids urine sample reported a few white blood cells. I am humbled by the commitment shown by all involved towards ensuring that my kid is safe and is not harmed by an experimental drug.

One year gone. At least as a community we know that exon skipping process is helping the kids who do get the drug. That is why the whole process is continuing. I am just happy to be part of this process. A few more weeks and we start producing dystrophin.

Sincerly hope and pray that each one of us get onboard of this distrophin production business ASAP. This exon skipping drug has to succeed for all and in time. Everyone is trying to contribute in their own special way.

We are all grateful for every boy and family taking part in all the trials. I also hope my son will be accepted in the near future. I see it as the only way he can have access to this medication in the next 3+ years. I think that both GSK/Prosensa and our boys have something to gain by taking part in these trials and if we need to be in a placebo group for a year, travel to have saline injections every week and biopsy the boys only to so the data can be analyzed and hopefully get this drug approved, then so be it. I don’t think any of us takes part in a trial w/o hoping that their son will benefit from this along the way. After all we are fighting to save our son lives and not just to advance drugs for the next generation.

What I find quite unethical (besides the fact that we biopsy the boys in placebo and inject them every week with saline although we do know that they do not produce any dystophin and we do know historically that they decline… of course I understand that they need to collect data on these boys so I accept it) is the fact that they need years and years of data AFTER the trial ends. If one wants to see if the strength is maintained for 2 additional years then the doctors can report it, no need to collect 2 years of additional data just to prove it to the scientific community, while boys are dying every day from Duchenne. What happens to the boys not allowed to participate in these trials? The ones that they decided not to select? Is it OK to let them decline and die while they collect 2 years of additional data? We are always talking about the boys participating in the trials, but there are hundreds not allowed to b/c they do not meet certain criteria, walk too fast, too slow, cannot rise from the floor independently etc. Is it fair to have them wait additional years if this drug is proven effective after the 1 year trial?

While I don't wish to descend into despair, I for one have zero confidence in western biopharma to help this generation of boys, for the exact reason Ofelia states - they are just too slow. Years to assess data on a few dozen boys? Years to decide whether to continue a treatment that shows some adverse events? It's absurd.

And spare me the stale old argument about slow = safe. We know that's not true for our boys.

The “slow=safe” argument is ridiculous and no well informed person believes it at this point. It just sounds like someone who doesn’t have any idea about drug development would use to scare us off.

Look for example at these drugs approved in 6 months or less, Gleevec is one of them. They all have side effects, Gleevec does, I am sure the rest do:

http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDeve...

http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDeve...

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