Ok - so I am hoping that someone can help me with how to be lined out for a clinical trial.  If you recall any articles on the subject - that would be helpful info. Thank you. Char Burke

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If you know of a trial you are wanting to be part of, a geneticist, neurogeneicist, neurologist, virtually any specialist can sponsor you to be part of the clinical trial if they feel you are eligible. 

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Char--if your son is entered into Duchenne Connect you will receive all the notifications about new trials and you may receive an invitation to be screened if the sponsors (academic group or company) go through Duchenne Connect directly.  You can always check www.clinicaltrials.gov and search for "Duchenne muscular dystrophy" to pull up listings of all the trials--you can even sort by the ones that are actively recruiting.  Those trial announcements will contain contact information for people who think their sons' might be eligible.


In general, most trials do allow the boys to be on steroids, but request that the dose/regimen be stable for some period before starting the study.  You can read through the eligibility and exclusion criteria in the clinicaltrials.gov listings.  Some trials might exclude boys who have participated in other trials recently, for example.  Usually it comes down to whether your son meets the criteria to participate, whether or not you are comfortable with the risks and benefits associated with participation (the trial coordinators will go over all of that with you in detail during the informed consent process) and whether or not it's practical for you to participate--based on number of visits, how far you have to travel, length of study etc.


So, what might be good prospects for a duplication?  Summit PLC is getting ready to do its first Duchenne trial but they haven't yet decided what country(s) will be involved.  Pfizer is in the midst of a "healthy volunteer" study with a drug that blocks myostatin and they plan to move to a Duchenne study if all goes as planned.  Shire is still working out the bugs with it's muscle growth promoting drug (blocks myostatin and other proteins that normally slow muscle growth) and Tivorsan is also planning a phase I study in Duchenne with its utrophin upregulator.  I don't have time lines yet for any of these, but they are all at the stage where the next step is to test in Duchenne (or continue testing as the case may be).  They are all mutation-independent approaches.


Hope this helps!


I forgot to mention Halo therapeutics, which is planning a phase I for halofuginone, a drug that may block the formation of fibrosis (scar tissue) that gets built up when muscle tissue dies.  They are also lined up for a trial in Duchenne, perhaps within the next year.




Thanks Sharon - that helps understanding about how to prepare for trials.  Can you explain once Utrophin is upregulated, does it allow the DMD person to sustain the muscle they have or does it actually help with building muscle?  I know one of the benefits of utrophin is that doesn't require immune suppressant drugs...If you had to guess whether the pharmas would choose phase II candidates, would they be looking to boys that still had quite a bit of muscle or ambulatory or would that matter?  Would you also explain biglycan too?   I have our son logged into the Duchenne Connect registry and have gotten several emails about the various trials.  Thank you for your help. Char Burke

Hi Char:

I think all we can do is speculate about what upregulating utrophin might do....ideally it would allow the boys to build muscle but that might depend on the stage of progression when the drug is started.  I think most investigators are shooting for stopping or slowing progression at this point, but it's generally recognized that we will eventually probably need a "cocktail" of drugs to really have the best benefit in DMD.  For example, a drug that blocked myostatin could allow the muscles to regenerate while at the same time a drug that upregulated utrophin might stabilize the new muscle that is produced.  Those are the kinds of cominbations we are thinking will be used in the future.  But right now, until we do those human clinical trials, we don't know what kind of results to expect.


So, biglycan seems to work by recruiting more utrophin to the muscle cell membrane and concentrating it there.  Again, in theory, you could use a drug like the Summit drug to increase the amount of utrophin the cell is actually making and then biglycan to make sure all that utrophin gets to the cell membrane where it's needed in a sort of one-two punch.  We've funded Lee Sweeney's lab at the University of Pennsylvania to look at some of these combination approaches in mice to see if you get additive effects. 


Finally, as to who will be eligible for the next round of clinical trials (utrophin, myostatin inhbitors, fibrosis blockers etc), the conservative approach is to use the six minute timed walk as an endpoint because companies know the FDA is willing to accept it, which means that the boys would need to be ambulatory; however, the National Institutes of Health and the FDA are sponsoring a meeting this Spring focused on upper body measurements to encourage companies to develop these and use them in clinical trials. PPMD has funded a number of different ways to do measurements in the boys that do not require that they be ambulatory and we are probably going to fund another one in the next round of grants.  So, if we can get these "nonambulatory endpoints" validated before the next round of clinical trials start, maybe some companies would be wiling to try it and the boys wouldn't have to be walking to participate.  All speculative, but that's what we are hoping.



Sharon - thank you again for sharing your knowledge. I try and stay current but it's hard being a full time employee and mom! I hope you and your family have a great holiday and New Year...Char Burke


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