I just read in the on-line Quest Magazine for MDA, that Dr. Mendell's AVIPharma Exon Skipping trials are beginning in March of 2010. I signed up for Nicolas to be a part of the trials back in April and have every now and then emailed the coordinator to get updates. He did confirm that it would be taking place at that time, and right now they are getting their protocols together and should have more information in a couple weeks.

Here's my dilemma No. 1. We see Dr. Wong, and she too will be starting clinical trials for the Netherlands exon skipping drug trial (not the same as Dr. Mendell), but I have no timeframe from her of when her trials will begin. I was told last year that they would start summer of this year.... It's almost Christmas. I want something now and don't want to wait any longer.... first come first serve I guess.

My other dilemma is that my husband and I both work in Illinois and the trial is on-site in Columbus Ohio for 12 weeks, and then about 5 follow up visits, every couple weeks after that. This will of course cost me to lose my job, and in this economy, we absolutely cannot afford that. Of course my heart and thoughts are with my son and no matter what, if he is accepted into the trial, we will be there, job or no job. This is definitely more important, but I can't help but think about the future of not having a job... most likely having a home foreclose on us (we are definitely not wealthy), and living with relatives (really not close with alot of them), etc. etc. etc. Also, not having enough $$ to afford to buy Nicolas his deflazacort, and supplements, and not to mention still paying for this years visit to see Dr. Wong.
I guess I just wanted to throw my worries out there. Hopefully it's all for the best. Is anyone else up for the trials or know about Dr. Wong's???

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we will also take the "whatever comes first" approach, as austin is declining fast, we just prefer a phase 3, which is what i believe prosensa wuld be, and a longer trial vs 3 months, if both would start roughly the same time we'd go for prosensa

Michelle Gonzales said:
Apparently there is to be a Prosensa trial in Cincinnati. The problem is I was told this last year that it would start in summer of 2009, and to my disappointment it did not. I did not receive any further specifics from Dr. Wong, but I am not going to wait for what could be beneficial to my son and every other boy out there and fight over AVIPharm or Prosensa. Dr. Mendell's study seems to be coming along quicker and if Nicolas is accepted into his trial - I'll be there. I'd like to hear if anyone has heard from Dr. Wong on if protocols are being done, and what month of next year her trial is expected to take place.
the response was not really clear, if you message me your email i will forward it to you if you'd like

Ofelia Marin said:
Does he give an explanation of why he thinks exon skipping will not become an effective treatment (if approved)? What does he think would be a better alternative?

jenn said:
i know dr brian at mass general in boston seemed to not like exon skipping at all when i asked him if that hospital may be a site for avi or prosensa... so confusing for sure!

Laurie Paschal said:
I'm confused, too. We were there in August and she didn't say a word to us.

jenn said:
im confused, we are also dr wongs patients, and also set to hopefully take part in the trial at nationwide childrens...but dr wong never told us about her possible trial, in fact i believe she mentioned we would have choices, prosensa or avi, in the next year but no further info. if anyone has info on where prosensa will be doing their trials id love to hear it.
did she say what phase, or if it would be prosensa, or anything else?

Michelle Gonzales said:
I just received an email response from Dr. Wong who stated that trials are to begin in 2010, but no further specifics of when. As I said before, first come first serve.

jenn said:
i sent her an email, she usually gets back fairly quickly, or askes andrea to answer... i will let you know what she says, also, last april when we saw her i asked about exon skipping, and she told me 51 would not work for my boys, that we need 53, we have since learned from steve wilton and others that either should do it...maybe dr wong has other opinions, i know dr brian at mass general in boston seemed to not like exon skipping at all when i asked him if that hospital may be a site for avi or prosensa... so confusing for sure!

Laurie Paschal said:
I'm confused, too. We were there in August and she didn't say a word to us.

jenn said:
im confused, we are also dr wongs patients, and also set to hopefully take part in the trial at nationwide childrens...but dr wong never told us about her possible trial, in fact i believe she mentioned we would have choices, prosensa or avi, in the next year but no further info. if anyone has info on where prosensa will be doing their trials id love to hear it.
so, i looked back at an email from dr wong in october and she said, prosensa will also be doing trials in the us and europe so it appears you will have choices. still have no idea when, or where...wonder why prosensa is not as open with things as avi seems to be
Jenn, I do not think that Prosensa had a confirmed list of US sites in October since they just partnered with GSK in November and GSK is planning/paying for the trial. Even now, beginning of December, they might have discussions/visit sites but the list might not be finalized (?).

Michelle, did Dr. Wong actually reply that Cincinnati will be a site for Prosensa/GSK's trial? We did know from Prosensa/GSK press release that Phase III starts 2010. Phase III couldn't have started 2009 since Phase I/II extension is in progress in Europe as we speak and GSK just came onboard last month.

jenn said:
so, i looked back at an email from dr wong in october and she said, prosensa will also be doing trials in the us and europe so it appears you will have choices. still have no idea when, or where...wonder why prosensa is not as open with things as avi seems to be
I'm waiting to hear further from Dr. Wong. It is Prosensa, I was not told if Cinci was the site for the trials, but assume it will be. I asked Dr. Wong if she could give me more specifics as to when the trials may begin and if protocols are being put into place or if they are still at the beginning. I do agree that I'd go for prosensa if they were to give a firm timeframe since it is in phase 3 - but my son is 7 and right now, i cannot wait any more. He is on the decline and I can't watch him go through this knowing that he had a chance for one trial, while the other is on hold for now. They may say that phase 3 will begin in 2010 - but without that certainty, i'm not waiting around.
I understand...I am under the impression that both trials will start around the same time or close, but I do not have any firm information. I think we'll learn more during the next couple of months. FDA needs to give the approval for the trials, I do not think any of them has that at the moment.


Michelle Gonzales said:
I'm waiting to hear further from Dr. Wong. It is Prosensa, I was not told if Cinci was the site for the trials, but assume it will be. I asked Dr. Wong if she could give me more specifics as to when the trials may begin and if protocols are being put into place or if they are still at the beginning. I do agree that I'd go for prosensa if they were to give a firm timeframe since it is in phase 3 - but my son is 7 and right now, i cannot wait any more. He is on the decline and I can't watch him go through this knowing that he had a chance for one trial, while the other is on hold for now. They may say that phase 3 will begin in 2010 - but without that certainty, i'm not waiting around.
When Kevin Flanigan was still at the University of Utah, he told me that his clinic was going to be a site for Prosensa's US trial, but now that he's moved to Columbus, I suspect that trial will follow him there.
I'm not entirely sure, because I was still in shock at his news that he was moving, but I believe he said Utah was still going to be able to participate in trials. I don't know who has authority to make that decision (Prosensa, FDA, physicians at the site), but I've not given up hope. One thing is certain, there will be multiple locations in the U.S.

Either way, a 16 week study in any US location is completely do-able for us. Either I take a personal leave, or my SAHM wife goes.

Paul Cliff said:
When Kevin Flanigan was still at the University of Utah, he told me that his clinic was going to be a site for Prosensa's US trial, but now that he's moved to Columbus, I suspect that trial will follow him there.
I think that Pat Furlong said in one of her summaries that there will be 12-13 sites in the US for Prosensa/GSK's trial.

Jerry Dallapè said:
I'm not entirely sure, because I was still in shock at his news that he was moving, but I believe he said Utah was still going to be able to participate in trials. I don't know who has authority to make that decision (Prosensa, FDA, physicians at the site), but I've not given up hope. One thing is certain, there will be multiple locations in the U.S.

Either way, a 16 week study in any US location is completely do-able for us. Either I take a personal leave, or my SAHM wife goes.

Paul Cliff said:
When Kevin Flanigan was still at the University of Utah, he told me that his clinic was going to be a site for Prosensa's US trial, but now that he's moved to Columbus, I suspect that trial will follow him there.
My son has a deletion of 51 and needs exon 50 skipped. Will this upcoming study in March benefit him? If so, who do I need to contact? Thanks so much. Happy holidays to all of you!

Ofelia Marin said:
I think that Pat Furlong said in one of her summaries that there will be 12-13 sites in the US for Prosensa/GSK's trial.

Jerry Dallapè said:
I'm not entirely sure, because I was still in shock at his news that he was moving, but I believe he said Utah was still going to be able to participate in trials. I don't know who has authority to make that decision (Prosensa, FDA, physicians at the site), but I've not given up hope. One thing is certain, there will be multiple locations in the U.S.

Either way, a 16 week study in any US location is completely do-able for us. Either I take a personal leave, or my SAHM wife goes.

Paul Cliff said:
When Kevin Flanigan was still at the University of Utah, he told me that his clinic was going to be a site for Prosensa's US trial, but now that he's moved to Columbus, I suspect that trial will follow him there.
from what iwas told is is a trial of the skipping of exon 51, so its not what you need, however, at the conference in london exon 50 was mentioned as a target for a trial, i think by prosensa but i could be wrong. make sure you are in the duchenne connect data base, that is how we were contacted for the march trial

Julie Hathaway said:
My son has a deletion of 51 and needs exon 50 skipped. Will this upcoming study in March benefit him? If so, who do I need to contact? Thanks so much. Happy holidays to all of you!

Ofelia Marin said:
I think that Pat Furlong said in one of her summaries that there will be 12-13 sites in the US for Prosensa/GSK's trial.

Jerry Dallapè said:
I'm not entirely sure, because I was still in shock at his news that he was moving, but I believe he said Utah was still going to be able to participate in trials. I don't know who has authority to make that decision (Prosensa, FDA, physicians at the site), but I've not given up hope. One thing is certain, there will be multiple locations in the U.S.

Either way, a 16 week study in any US location is completely do-able for us. Either I take a personal leave, or my SAHM wife goes.

Paul Cliff said:
When Kevin Flanigan was still at the University of Utah, he told me that his clinic was going to be a site for Prosensa's US trial, but now that he's moved to Columbus, I suspect that trial will follow him there.

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