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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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Permalink Reply by wainaina muiruri on May 3, 2012 at 12:20pm

This goes to GSK in regards to the clinical trials they have going.Is it possible for your company to consider running some of the trials in Africa?After all you have had a very strong presence in the African market for some decades and it would only be fair for you to do so.Thank you and hope you will address my query.

Regards

Wainaina Muiruri

Nairobi,Kenya

Permalink Reply by David on May 3, 2012 at 7:22pm

I would be grateful if you asked why nobody seems to run trials that include non-ambulatory boys? The technology and techniques to measure clinical benefit without a 6-minute walk test surely exist.

Permalink Reply by Tammy Henegar on May 7, 2012 at 1:28pm

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.

StaffPermalink Reply by Kathi Kinnett on May 7, 2012 at 2:49pm

Thanks everyone for their questions. I will be sure to include them, in some way, in the discussion at the annual conference. Keep the questions coming :)

Permalink Reply by Jason Darienzo on May 7, 2012 at 3:50pm

There are 2 different compounds to skip exons. Antisense Oligonucleotides (AONs) by Prosensa and Phosphorodiamidate Morpholino Oligomers (PMOs) by AVI. AONs seem to stay in the body longer, work a bit faster but have some side effects. PMOs seem to have no side effects but take awhile to start taking effect(at least 24 weeks).

I think its was good both started with exon 51 trials because to see which works better, if there are differences side effects, and back up if one fails. But now they are starting to do different ones.

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.

Permalink Reply by Tammy Henegar on May 7, 2012 at 3:59pm

Jason, I do know that they have been moving on to others, but it just semed a little confusing as to why both to start on 51. I know there is a lot going on right now, more then ever before.. it just isn't quick enough for our boys.

Thank you for the information on the compunds. I appreciate it.

Permalink Reply by Jason Darienzo on May 7, 2012 at 4:21pm

Tammy, I'm 32 with DMD. I want it fast too but the science needs to be right. Your son is young and has time to benifit from all these drugs in the pipeline.

Permalink Reply by danielle on May 7, 2012 at 7:39pm

What I want to know is why there's no one trying to fix exon 45 I have been looking for clinical trails to help this

Permalink Reply by Keith Van Houten on May 7, 2012 at 8:01pm

Because exon 51 is the most commonly needed skip. It's the largest market. It helps 60% more boys than the next most commonly needed exon skip - which is exon 45, by the way.

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.

Permalink Reply by Jason Darienzo on May 7, 2012 at 8:36pm

If you have exon 45 deletion you need to skip 44. Prosensa is in phase I/II trials with Pro 44. If you need to exon 45 skiped thats up next in pre-clinical at AVI and Prosensa.

"Which exon should be skipped?" http://prosensa.eu/patients-family/duchenne-muscular-dystrophy/faq/...

danielle said:

What I want to know is why there's no one trying to fix exon 45 I have been looking for clinical trails to help this

Permalink Reply by Cheri Gunvalson on May 8, 2012 at 9:11am

http://www.sciencedaily.com/releases/2012/05/120504110554.htm

Hi Kathi,

If you have anyone from this study presenting please ask when they think this might be going to human trials. Thanks

Permalink Reply by David on May 8, 2012 at 10:40am

I have another question:

Since the natural history of Duchenne is well-known, why rely on placebo-controlled short-term studies? Seems to me that for any study that lasts several years, it would be impossible for the "placebo effect" to confound the results.

-David