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Permalink Reply by Andrea Cleary on June 17, 2013 at 10:58am

Dear Donna,

Your son would need to walk, non-stop, for 6 minutes. There are even criteria for the distance they would need to walk (lower acceptable limit) to be included in the trial, because if they cannot walk that # of meters required, it means they are most likely already in a period of decline and then the drug would not show as much of a benefit, risking that the drug not be approved. Same reason they have age limits. Since lots of boys improve over the age of about 4-7, often they do not want to include them because it cannot be proven whether the boy is improving due to the medication or just because they would have anyway. Extremely frustrating when we would all do anything for any improvement for our kids.

Andrea

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Permalink Reply by Donna Cicardo on June 17, 2013 at 11:43am

Thanks Andrea, so if they are in this decline, a drug is approved, our boys in decline will they get the drug will they.  sometimes I feel as if they forget about the older boys.  I pray I am wrong but its just hard.  my son is trying to lose weight.  he needs to lose another 25-30 lbs.  maybe after the weight loss he may be strong enough to walk a full 6 minutes.  have you heard of anyone losing weight and then improving on walking.

Andrea Cleary said:

Dear Donna,

Your son would need to walk, non-stop, for 6 minutes. There are even criteria for the distance they would need to walk (lower acceptable limit) to be included in the trial, because if they cannot walk that # of meters required, it means they are most likely already in a period of decline and then the drug would not show as much of a benefit, risking that the drug not be approved. Same reason they have age limits. Since lots of boys improve over the age of about 4-7, often they do not want to include them because it cannot be proven whether the boy is improving due to the medication or just because they would have anyway. Extremely frustrating when we would all do anything for any improvement for our kids.

Andrea

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Permalink Reply by Ofelia Marin on June 17, 2013 at 1:46pm

They do exclude 'older' more advanced boys and recently younger ones (age 5-7) from trials. If the drug is approved, assuming that it is not approved only for ambulatory with an age limit (older than 7 for example), then all boys can have access and insurance should pay for the drug (in most cases). It is not known how these drugs will be approved. They are usually approved for the population they were tested in. It doesn't make any sense for Duchenne to require additional trials in non-ambulatory boys though, however it is not clear how this will work out.

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Permalink Reply by Andrea Cleary on June 17, 2013 at 4:19pm

Donna,

I was not trying to suggest what will happen once a drug is approved (thanks Ofelia), just why a non-ambulatory or in decline boy cannot be part of the trials when the 6MWT is used, based on pharmaceutical reasoning (not mine). This is what the Leclaire (mom Jenn McNary) case is about http://abcnews.go.com/Health/vermont-brothers-deadly-disease-healin...

Difficult to be forced to accept.

Andrea

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Permalink Reply by Donna Cicardo on June 17, 2013 at 5:11pm

Yes, thank Andrea,  I understand. It is just so hard, we were asked to be in studies but the said at first we were diagnosed BMD.  and we could not be in them because we were called BMD.  At the time my son was still walking a lot,  a lot more than 6 minutes.  Then everything changed one year ago, our doctor here in texas had misdiagnosed us and we then found out we have DMD by doing more testing. and have progressed as DMD.  We were contacted to again for the Talafadil study for this September.  they are going to be contacting us again in August.  She did not discuss how much of the 6MWT but only said they need 1000 boys.  on Clinicaltrails.gov it says must perform 6mtw but it did not say if you could not complete the walk you would be excluded.  I think we are going to focus on weight loss and see if at that time he can do the walk in our house and if he can then if they call for the study we will try.  I just hope all these studies we have been raising money is not going to be just for those that walk if the drugs are approved.  I have a feeling the FDA is going to say it is only approved for those who walk.  If our US Govt. has anything to do with anything, they will put so many restrictions on the drug like they do everything else.  Thanks Ofelia, I hope and pray you are right.
 
Andrea Cleary said:

Donna,

I was not trying to suggest what will happen once a drug is approved (thanks Ofelia), just why a non-ambulatory or in decline boy cannot be part of the trials when the 6MWT is used, based on pharmaceutical reasoning (not mine). This is what the Leclaire (mom Jenn McNary) case is about http://abcnews.go.com/Health/vermont-brothers-deadly-disease-healin...

Difficult to be forced to accept.

Andrea

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Permalink Reply by Tulika on June 17, 2013 at 6:29pm

I know that GSK trial has a few kids that stopped walking during the duration of the trail. They are still on the trial and part of the approval process even though they have stopped walking. 6MWT is the primary measurement criterion but I dont think that approval will be done only for ambulatory kids.

AVII deck in the past years had said that revenue pipeline was in few 100K per kid per year. Insurance companies will have their reasons on approving such costs with no apparent benefit for non ambulatory kids. I think this is where PRO scores over AVII since PRO costs are much much lower.

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