Hello,
I just got off the phone with Lindsay, the clinical trials coordinator for Dr. Mendell. She asked me to post that anyone whose son is between 5-15 who would benefit from skipping 51 who is interested in participating in a systemic trial should contact her at arnottl@ccri.net.

The trial will have a placebo arm. It will take place in Columbus. I don't know the dosage.

Take care, all,
Mindy

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This was asked at the conference this weekend. They will have to find a way to make sure only one of the exons is skipped and not both.

Amanda Rudd said:
Does this only work with deletions? My son has a duplication of 45 will he be an eligible canidate?

cheryl cliff said:
Time to start packing for the UK. Recently we asked Dr Flanigan's opinion about who will come in first - Prosensa or AVI. He said Prosensa. Looks like he might be right.

Thanks for bringing this tidbit to our attention Ofelia. Even tho it isn't great news we still need to know how things are coming along.
cheryl

Ofelia Marin said:
More delays...

http://www.avibio.com/duchenne_muscular_dystrophy.php

In addition, AVI has now started the comprehensive series of preclinical tests necessary before a US study with AVI-4658 can be undertaken. The results from these studies will need to be reviewed by the FDA before a US study can start. It is hoped that the US study could start in early 2010.

AVI is advancing a second product, targeting exon 50, and has now started a similar series of preclinical studies with AVI-5038. While these studies are underway, AVI anticipates having discussions with the Regulatory Authorities about plans to start clinical work with AVI-5038. This additional regulatory dialog could help with planning for candidates to skip other common deletions. The early results from our PMO program (with AVI-4658) should help us to streamline this PPMO program; we hope to start a clinical study in mid 2010.

AVI is examining other target deletions with the hope of developing the best candidates for treating these target deletions.
Hi Laurie,

Were there any other news/results from the Prosensa and AVI trials? Anything about timing?

Thanks

Laurie Paschal said:
This was asked at the conference this weekend. They will have to find a way to make sure only one of the exons is skipped and not both.
Timing for the US is going to depend on the FDA. If they'll allow the trials here, I'm assuming without any additional safety studies, etc, then 2010 could be a very good year. If not, then the trials could be completed and the drugs have final approval in the other countries in 2012.

Ofelia Marin said:
Hi Laurie,

Were there any other news/results from the Prosensa and AVI trials? Anything about timing?

Thanks

Laurie Paschal said:
This was asked at the conference this weekend. They will have to find a way to make sure only one of the exons is skipped and not both.
So, ultimately it appears as though living or moving (sounds good but is it possible) is the best way to get our older sons into clinical trials for exon skipping.

I realize they want patients to live near clinical trial sites for practical reasons. I understand trials aren't convenient or pretty. But, it sure beats death and some of our boys can't wait until the FDA gets up to speed.
cheryl
Thanks Laurie. Where there any research news this conference compared with last year's. We already knew that utrophin upregulation trial was delayed for next year, same with exon skipping. Any progress with anything else or only the delays?
We have not heard anything about Justin Fallon's Biglycan in a while...that was supposed to advance to clinics next year (at least that is what they said last year).



Laurie Paschal said:
Timing for the US is going to depend on the FDA. If they'll allow the trials here, I'm assuming without any additional safety studies, etc, then 2010 could be a very good year. If not, then the trials could be completed and the drugs have final approval in the other countries in 2012.

Ofelia Marin said:
Hi Laurie,

Were there any other news/results from the Prosensa and AVI trials? Anything about timing?

Thanks

Laurie Paschal said:
This was asked at the conference this weekend. They will have to find a way to make sure only one of the exons is skipped and not both.
Hi Jenn,

I have no contacts at AVI or Prosensa. You could e-mail Muntoni. I'm with you tho, staying abroad for a year or so wouldn't be fun but the alternatives stink.

The where part of your question is easy to answer, London. And, from what I've heard obtaining a flat there isn't all that difficult, just expensive given currency conversion and all. Are you seriously thinking of trying to do this? I too believe in exon skipping and would be happy to help in any way I can, if you wish. I'd love to see an American boy enrolled in an exon skipping trial. We have to wait longer since we need to skip 45 and who knows when that number will come up.
cheryl

jenn said:
do you have a lead on who to contact and where theoretically we would want to be with our boys ( and when ) in order to be a part of a trial? also is relocating allowed in this circumstance? we are really in no position to up and move so far away, but i do believe in this new drug and just don't want to sit and wait until austin is not a candidate anymore. we could possibly rent something for the duration of the trial.

cheryl cliff said:
So, ultimately it appears as though living or moving (sounds good but is it possible) is the best way to get our older sons into clinical trials for exon skipping.

I realize they want patients to live near clinical trial sites for practical reasons. I understand trials aren't convenient or pretty. But, it sure beats death and some of our boys can't wait until the FDA gets up to speed.
cheryl
Hi Jenn,

During exon skipping trials some boys recieve meds others not. You will have no way to know which your son(s) will be recieving. There is a 50/50 chance for each and you must decide to take that chance if you enroll. Even still, those odds are better than waiting and watching.

If they know you are from the US they probably won't give you a positive response for enrollment in the current trial. You might want to consider looking into where they are going to conduct the current trial. I believe its going on in several UK locations. I would then secure an address of a place to stay in that location. Then try to enroll. Or, just tell them you are in the process of obtaining a place to rent for a year or so and see if they will work with you.
cheryl



jenn said:
cheryl, i am commited to doing anything i can to include austin in this trial. it is the first time i have felt hopeful in a long time. he is not going to be walking much longer and im afraid the columbus trial will be too late. his little brother max is doing much better, but id enroll both if we could be sure we'd get the drug.my mother has already said she'd take a year off if needed( my husband and father will need to keep running our family business here if we are going to be able to afford the trip. i have sent an email to the address listed at the trial site, if we get a possitive response, london here we come!
as for when 45 will come up, im sure as soon as these trials have good results they will be adding every number to the list. i'd love your support, not sure how yet, but i'll keep you posted on the progress. thanks, jenn
cheryl cliff said:
Hi Jenn,

I have no contacts at AVI or Prosensa. You could e-mail Muntoni. I'm with you tho, staying abroad for a year or so wouldn't be fun but the alternatives stink.

The where part of your question is easy to answer, London. And, from what I've heard obtaining a flat there isn't all that difficult, just expensive given currency conversion and all. Are you seriously thinking of trying to do this? I too believe in exon skipping and would be happy to help in any way I can, if you wish. I'd love to see an American boy enrolled in an exon skipping trial. We have to wait longer since we need to skip 45 and who knows when that number will come up.
cheryl

jenn said:
do you have a lead on who to contact and where theoretically we would want to be with our boys ( and when ) in order to be a part of a trial? also is relocating allowed in this circumstance? we are really in no position to up and move so far away, but i do believe in this new drug and just don't want to sit and wait until austin is not a candidate anymore. we could possibly rent something for the duration of the trial.

cheryl cliff said:
So, ultimately it appears as though living or moving (sounds good but is it possible) is the best way to get our older sons into clinical trials for exon skipping.

I realize they want patients to live near clinical trial sites for practical reasons. I understand trials aren't convenient or pretty. But, it sure beats death and some of our boys can't wait until the FDA gets up to speed.
cheryl
Hi Jenn,
I tried to get into the London trial when the systemic phase started, and was told they were not taking American boys - not at Newcastle, either.

Always worth trying again, though...Let me know how it goes.
Mindy
mindy, thanks, i'm sure you are right, i wonder why they wont consider american boys?
it's always an uphill battle... still, it's worth a try i suppose. jenn

Mindy said:
Hi Jenn,
I tried to get into the London trial when the systemic phase started, and was told they were not taking American boys - not at Newcastle, either.

Always worth trying again, though...Let me know how it goes.
Mindy
They told me at the time that it was a safety trial only - didn't expect efficacy, and didn't want people to move from home just to verify safety.
does that mean that they may consider an american child for the current trial? i got an "out of office reply" so i hope they will reply when they are back, it said july1st.

Mindy said:
They told me at the time that it was a safety trial only - didn't expect efficacy, and didn't want people to move from home just to verify safety.
Usually the saftey trial is done on healthy adults rather than on children.

jenn said:
does that mean that they may consider an american child for the current trial? i got an "out of office reply" so i hope they will reply when they are back, it said july1st.

Mindy said:
They told me at the time that it was a safety trial only - didn't expect efficacy, and didn't want people to move from home just to verify safety.

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