PTC 124 TRIAL
Is there anyone who has gone through the trial screening or even potentially getting the drug in the 2b trial,if so how long have you been in the trial and please share your thoughts.It would be helpful for us and many more who are waiting to get in the trial.
Thankyou
Thankyou
Replies to This Discussion
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Permalink Reply by LarryW on
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Yes, it would be nice to hear from anyone that would be going through the new trials. There is nothing that gives us progress reports on how far along they are on the 2b trials.
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Permalink Reply by MicahsDaddy on
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if you haven't already, sign up in the registry for DuchenneConnect. It is a database that has all your information so the professionals can find people who may qualify for clinical trials. Here is a thread explaining what it is:
http://community.parentprojectmd.org/forum/topic/show?id=1187424%3A...
Also, at the PPMD conference, they said there is still room in the trial for candidates, as long as the criteria is met. Here's a link to ClinicalTrials.gov where you can check for eligibility: http://www.clinicaltrials.gov/ct2/show/NCT00592553?term=duchenne&am...
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Permalink Reply by Donnie and Linda Owens-Jacob Owens on
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we take jacob next wednesday to cincy for a muscle biopsy and more prelim test. he should start taking the medicine(or placebo) when we go back the following wednesday.
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My grandson started the drug (or placebo) 3 weeks ago. It took several months of screening, testing and waiting to get this far. There doesn't seem to be any change of any kind so.
I also would like to hear from anyone who went through the 2A trails to hear of their experiences.
At the hospital my grandson goes to, they had trouble finding boys that met all the criteria and could participate.
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Permalink Reply by Sharyn Thompson on
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We tried to get on the trial, but James has just turned 4 (in May) so they considered him too young. We actually fought hard to get him included, but they were very strict about their criteria. Hopefully, they still won't have the numbers by the time he is 5 and we may get him on the trial early next year, as I hear they are still trying to recruit boys.
I would also be interested to hear from anyone whose son participated in the last trial.
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Donnie and Linda Owens-Jacob Owens said:we take jacob next wednesday to cincy for a muscle biopsy and more prelim test. he should start taking the medicine(or placebo) when we go back the following wednesday.
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Permalink Reply by Angela on
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My son participated in the 2a trial 2 1/2 years ago. Jack had his biopsy, etc. last week for the 2a extesion and will go back to Utah next week to start the drug. If you have questions that I can help with, please contact me directly.
Ang
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Permalink Reply by neeraj on
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Hi Michelle
I am not sure where you got that from about non ambulant kids participating in the ptc trial,I know ptc is very tough on the set criteria and only currently taking ambulant kids.My hope is the drug works and benefit the boys with nonsense mutation and other drugs come out soon to benefit everyone.Although i can do with everyone involved in running the trial to not take so long to start everything.We are in UK and trial centre we will go to has not even started recruiting and we have been waiting since march this year.
How old is your son and does he have point mutation.
Regards
Neeraj
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Permalink Reply by Ellen Wagner on
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Michelle,
The confusion might stem from the two trials that are happening simultaneously. The 2b trial does require boys to be ambulatory but the 2a extension trial requires that the boys participated in the original 2a trial. At the time of the origianl trial all boys had to be ambulatory but to enter the extension 2a they may no longer be walking and are still eligible because they were in the first trial. I hope this helps. My son Tim was in the first trial and we are about to start the screening process for the 2a extension in Cinn. We saw great benefit the first time and are very hopeful for this new trial. The blood work and biopsies were difficult but Tim made it through and is ready to do it again.
Ellen
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Permalink Reply by Angela Ryan on
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Our son Jacob (age 9) started the 2b trial in Philadelphia 3 weeks ago - that is when he first got the drug/placebo. It was a little difficult getting used to the dosing three times a day as prior to this he only took meds in the morning. We are in a routine now and everything is going well. We hired a sitter to come into our house to watch our preschooler and so that Jacob could come home after school. He takes the midday dose when he gets home from school - which is why I wanted the sitter. This way I don't have to count on the school to do the dosing and return the empty packets. It makes it easier on Jacob too because he doesn't have to be singled out to take medication at school. We have seen no change yet: but today he seemed like he was going to jump out of his skin he was so hyper - don't know why that was. He had bloodwork done this morning and he gets upset when that happens so that could be why he was so antsy. We'll have to see how he is tonight.
So, the study is going well from our perspective.
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i am from france and my son has nearly finished the tested of inclusion; he will begin to take the medecines on the 10th of octobre;
best
françoise
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