Any information regarding Sildenafil as DMD treatment?

Last June at PPMD conference in Denver I learned of a trial involving Sildenafil, aka Revatio. My notes say the trial was to be held at University of Seattle, although the official posting says Johns Hopkins in Maryland.


Can PPMD share any updates on the progress of this program? My interest was piqued after reading about more recent studies with Aminophylline in zebrafish that seem to have a significant impact. Aminophylline acts in similar manner to Sildenafil.


Thanks as always for any inside info you may have. Searching through Google, etc. has not turned up much.



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Hi David--as you probably know there are clinical studies underway testing both sildenafil and tadalafil (see and for information) and we will be making an announcement about a study in younger boys soon.  We definitely see this category of drugs as "low hanging fruit"--things that can be tried in the clinic now while the more exotic therapies are still in development.


Best Wishes,


PPMD Research Director

Actually I missed the fact that PPMD was funding the Revatio trials. That's great information, thank you. But I'm still unclear about when to expect results.

The link says Study Start Date: September 2010.

PPMD posting says: In the first part of this two-part study one group will receive the drug and the other will receive placebo. After six months, the study will be unblinded and continue for another six months, 


Sept 2010 + 6 months would be March or April 2011, by my calendar. Is that accurate? Can you provide a more accurate timeline for the completion of the "first part"? Of course I know these are estimates, but it's better than nothing.



Hi David--we aren't actually funding the Revatio study at Johns Hopkins, but we will be funding a study in younger boys (both Cialis and Revatio in a head-to-head comparison).  That announcement will be out soon.  I don't know if the Revatio study is on schedule still or not--I know they had some trouble with recruitment, also true of the Tadalafil (Cialis) study. I can try to find out from Kathy Wagner and get back to you.



This is wonderful news.  From what I understand, PDE5 inhibitors increase nitric oxide.  I cant help but wonder why these & other NO donors haven't been tried sooner.  Seems they would be good in combination with steroids.  

Will these drugs will go thr same phases (I,II,III) like any new drug or will there be some shorter timeline and will availabe as therapy soon?


Great news - PPMD is backing some significant research in the area.


Amit - I believe we should expect a shorter timeline here too, I *think* that's what Sharon meant by "low hanging fruit."

Yes, these drugs are already approved so the timeline is shorter.  We will do a dose-finding study first and then a larger definitive study.  The press release should be out some time this week.

Thanks David and Sharon for the great news.
Sharon, What's the current state of Sildenafil/Revatio research? The Cedars-Sinai study is underway? It was strictly  for the ambulatory, correct? Will it move out to the non-ambulatory population next? Thanks!

Has anyone tried Revatio and got a good result?

We just received the consent forms for the trial on Tadalafil &Sildenafil.  Can anyone tell me something about it and what, if any, literature is out there about it?

If we participate in this trial, will it STOP us from possibly jumping ship to Sarepta's possible pivotal trial?

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