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If you had a chance to ask researchers, providers or industry any question at all about clinical trials, what would it be?

If you had a chance to ask researchers, providers or industry any question at all about clinical trials, what would it be?


PPMD’s 2012 Connect Conference in Fort Lauderdale is shaping up to be the most informative meeting we have hosted. This year, PPMD’s agenda will include a three part series on Clinical Trials. We will hear panel presentations from parents whose children are or were participating in trials, from clinicians who run the trials and from industry leaders themselves who develop the trials. These interactive panels will give us a glimpse into the future of Duchenne trials and provide new information we look forward to sharing with the community.


In anticipation of this panel format, we wanted to reach out to you – the Duchenne community – and find out the questions you would ask if you had a chance to ask researchers, providers or industry any question at all about clinical trials.


Reply with your question and we will do our best to submit them to these expert panels!

 

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Exon 44 is also in trials and several others to come: http://www.prosensa.eu/

I think, at this point, the big question is if these exon skipping drugs work at all. They need to show that at least one is safe and effective. So far that has not been proven.

Tammy Henegar said:

I would love to know why 2 differenct pharmaceutical labs have to both concentrate on the same exon, such as what is happening with the exon 51 trial?  Why can't they all work together, and agree to work on 2 different exons to try to further this a long a lot faster.  Time is not something that none of us necessarily have, and we all know that when they say it will be soon, it's not soon enough.

I appreciate everyone's input into this discussion. I think that each of the industry representatives will speak to their individual trial progress at the annjal meeting and that all of the above quesitons will be addressed and, hopefully, answered. What I am interested in for the panels are questions that you may have regarding the actual clinical trials. I think I have the gist of your questions: how do companies decide which compands to put into trial (51 vs 45), what is involved in setting up a clinical trial, how do they decide on locations (US vs Africa), who should be included (ambulatory vs non-ambulatory)?

Do you also have questions of clinicians who run the actual trials?? Keep them coming :)

My question is for SUMMIT. Considering that, this new PH1 trial will be successful, are they planning for PH 2 trial to be in U.S ?

I've written this question a dozen times in my head and most of the time it comes out with angry expletives. This is toned-down version:

Question for Shire/Acceleron: How much longer do you expect the DMD community to wait on some information regarding the future of ACE-031? It's now been over a year already since the study was terminated, and nothing has been made public other than re-assuring but vague comments that they are committed to the program. If they won't share WHAT they are doing, can't they share WHEN?

 My son has an insertion in exon 29 causing a frameshift. From what I can tell, Utophin drugs are the only things out there for him. My question is , if utophin drugs work in all the boys, why are we not concentrating our efforts on that?

Hi Bob: Your son might benefit from efforts to block the myostatin pathway (increases muscle growth and strength) that are underway by Shire and Pfizer or from new drugs in development by Catabasis and Reveragen that are designed to take the place of prednisone without the side effects, in addition to utrophin up-regulating strategies. So I think there are lots of things in development out there that are not "mutation-specific." And that doesn't even get into already approved drugs like Tadalafil and Eplerenone that are also in testing and may provide significant results as well--a drug doesn't have to be new to be effective.

But, your question is a philosophical one, I think, and that is why aren't we only funding strategies that will benefit all boys and the answer is that we don't know up front what's going to work--we don't think at this point it makes sense to put all our eggs in one therapeutic basket so PPMD funds the development of a variety of different therapies including mutation-specific and non-mutation specific strategies. There are very real benefits that might be seen from an exon-skipping approval for everyone, including those without skippable mutations. My last blog addressed some of these things (http://community.parentprojectmd.org/profiles/blogs/beyond-exon-51).

Sharon
(PPMD Research Director)

Is there anyway to influence where a trial is run?  For example, we have 4 boys in our family all with a deletion of exon 45.  All still ambulatory.  Would the drug companies be interested in this "cluster" even if its in a relatively low population area, and in Canada?

So, actually, drug companies do look for geographic "clusters" when they are planning trial sites, but they also have to take into account the experience and capabilities of the local center for running trials.  So you guys are looking for the exon 44 skip which Prosensa has in testing now...it might be worth letting them know that you guys are out there. 

Sharon

Sharon any new info on exon 44 skip trial in Europe

Estimated Primary Completion Date on clinicaltrials.gov is August 2012

Hi Jason--I suspect that they are planning to report on this study at the upcoming World Muscle Society meeting in Perth, Australia.  I'm not going this year, but Dr. Bob McDonald, our Board Chair is going and is going to report back for us.  I'll put this on his hit list of things to follow (it's probably there already).  That meeting is the week of October 8th.

Sharon

My concern goes to Ataluren. My son got Stop Codon in exon 16. Ataluren is waiting for approval to Europe then USA. So, how about other countries (we are living in Vietnam). If it is approved in EU, can we get from there ?

Thank you.

Thanks for your input ... much appreciated



Sharon Hesterlee said:

Hi Bob: Your son might benefit from efforts to block the myostatin pathway (increases muscle growth and strength) that are underway by Shire and Pfizer or from new drugs in development by Catabasis and Reveragen that are designed to take the place of prednisone without the side effects, in addition to utrophin up-regulating strategies. So I think there are lots of things in development out there that are not "mutation-specific." And that doesn't even get into already approved drugs like Tadalafil and Eplerenone that are also in testing and may provide significant results as well--a drug doesn't have to be new to be effective.

But, your question is a philosophical one, I think, and that is why aren't we only funding strategies that will benefit all boys and the answer is that we don't know up front what's going to work--we don't think at this point it makes sense to put all our eggs in one therapeutic basket so PPMD funds the development of a variety of different therapies including mutation-specific and non-mutation specific strategies. There are very real benefits that might be seen from an exon-skipping approval for everyone, including those without skippable mutations. My last blog addressed some of these things (http://community.parentprojectmd.org/profiles/blogs/beyond-exon-51).

Sharon
(PPMD Research Director)

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