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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well, im pretty sure that the safety trial in the UK was on kids ages 12-17 and it has already been completed, the trial scheduled to begin there in january is a dose escalation efficacy trial on kids ages 5 to 15.

Cheri Gunvalson said:
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.
Hi Jenn,

Yea, keeps my head spinning too! I do think they might take American boys who already live in their trial areas and those areas are spreading. I think they want to expand their trials and increase the amount of boys in it. Currently at ClinicalTrials.gov they list AVI seeking enrollment for the systemic trial currently underway, unless I read it wrong. And, one doesn't actually have to move to the UK, one simply requires an address close to a trial site (secure a rental and move after being accepted). Still they will probably require some convincing even if your sons fit their criteria. And then your son may/may not receive the meds vs placebo during. Not going to be easy, but then as you said before nothing regarding DMD is.

Should we do something about this collectively?

cheryl
I actually have a cousin living in london for another year or so...if I hear anything from the trial folks I can provide them a local address,of course we'd rent once we got there, but at least that's a start. Yes, I think we should make our son's known as candidates for trials, older boys won't have a chance by the time trials hit the us. I don't know how I feel about the chance that austin won't get the drug, but I KNOW he won't if we stay here and wait. Why would they use a placebo if the safety study is done, can't they just do a natural course study of duchenne?
My son will be 7 in July. He has a deletion on exon 51. I don't think he would benefit from this trial. Am I correct? I just returned from the conference and the news about exon skipping is very exciting. Kathy Busby from the UK stated that trials would be taking place in the U.S. I think my son needs 50 or 52 skipped. Blessings to all of you.

jenn said:
I actually have a cousin living in london for another year or so...if I hear anything from the trial folks I can provide them a local address,of course we'd rent once we got there, but at least that's a start. Yes, I think we should make our son's known as candidates for trials, older boys won't have a chance by the time trials hit the us. I don't know how I feel about the chance that austin won't get the drug, but I KNOW he won't if we stay here and wait. Why would they use a placebo if the safety study is done, can't they just do a natural course study of duchenne?
Does anyone know how I can get information on Charley's fund? Maybe a phone number or email. Thanks so much.

Julie Hathaway said:
My son will be 7 in July. He has a deletion on exon 51. I don't think he would benefit from this trial. Am I correct? I just returned from the conference and the news about exon skipping is very exciting. Kathy Busby from the UK stated that trials would be taking place in the U.S. I think my son needs 50 or 52 skipped. Blessings to all of you.

jenn said:
I actually have a cousin living in london for another year or so...if I hear anything from the trial folks I can provide them a local address,of course we'd rent once we got there, but at least that's a start. Yes, I think we should make our son's known as candidates for trials, older boys won't have a chance by the time trials hit the us. I don't know how I feel about the chance that austin won't get the drug, but I KNOW he won't if we stay here and wait. Why would they use a placebo if the safety study is done, can't they just do a natural course study of duchenne?
An idea for consideration if your sons are nonambulatory when the trial starts is when our son Jacob was denied PTC 124 the head of the neuro pharm div of the FDA recommended a protocal exception. A child in a trial with a protocal exception is treated the same as all the others but they do not include outcome measures when they drug company send its report to the FDA. Unfortunately PTC would not go along with the FDA. The FDA actually encourages expanded access ie compassionate use or a protocal exception in rare diseases such as duchenne so there are more on the drug in a controlled setting before it goes to market. I found the FDA to bend over backwards trying to help Jacob get on PTC 124. Feel free to email me if you have any questions, cgunval@gvtel.com

Julie Hathaway said:
Does anyone know how I can get information on Charley's fund? Maybe a phone number or email. Thanks so much.

Julie Hathaway said:
My son will be 7 in July. He has a deletion on exon 51. I don't think he would benefit from this trial. Am I correct? I just returned from the conference and the news about exon skipping is very exciting. Kathy Busby from the UK stated that trials would be taking place in the U.S. I think my son needs 50 or 52 skipped. Blessings to all of you.

jenn said:
I actually have a cousin living in london for another year or so...if I hear anything from the trial folks I can provide them a local address,of course we'd rent once we got there, but at least that's a start. Yes, I think we should make our son's known as candidates for trials, older boys won't have a chance by the time trials hit the us. I don't know how I feel about the chance that austin won't get the drug, but I KNOW he won't if we stay here and wait. Why would they use a placebo if the safety study is done, can't they just do a natural course study of duchenne?
thanks cheri, i hadn't even considered this.

Cheri Gunvalson said:
An idea for consideration if your sons are nonambulatory when the trial starts is when our son Jacob was denied PTC 124 the head of the neuro pharm div of the FDA recommended a protocal exception. A child in a trial with a protocal exception is treated the same as all the others but they do not include outcome measures when they drug company send its report to the FDA. Unfortunately PTC would not go along with the FDA. The FDA actually encourages expanded access ie compassionate use or a protocal exception in rare diseases such as duchenne so there are more on the drug in a controlled setting before it goes to market. I found the FDA to bend over backwards trying to help Jacob get on PTC 124. Feel free to email me if you have any questions, cgunval@gvtel.com

Julie Hathaway said:
Does anyone know how I can get information on Charley's fund? Maybe a phone number or email. Thanks so much.

Julie Hathaway said:
My son will be 7 in July. He has a deletion on exon 51. I don't think he would benefit from this trial. Am I correct? I just returned from the conference and the news about exon skipping is very exciting. Kathy Busby from the UK stated that trials would be taking place in the U.S. I think my son needs 50 or 52 skipped. Blessings to all of you.

jenn said:
I actually have a cousin living in london for another year or so...if I hear anything from the trial folks I can provide them a local address,of course we'd rent once we got there, but at least that's a start. Yes, I think we should make our son's known as candidates for trials, older boys won't have a chance by the time trials hit the us. I don't know how I feel about the chance that austin won't get the drug, but I KNOW he won't if we stay here and wait. Why would they use a placebo if the safety study is done, can't they just do a natural course study of duchenne?
I don't know if they are using placebos in the current trial, you could find out by checking clinicaltrials.gov and they would have more information. I suspect you have already registered your sons in DuchenneConnect? If not that is good to start with.
cheryl

jenn said:
I actually have a cousin living in london for another year or so...if I hear anything from the trial folks I can provide them a local address,of course we'd rent once we got there, but at least that's a start. Yes, I think we should make our son's known as candidates for trials, older boys won't have a chance by the time trials hit the us. I don't know how I feel about the chance that austin won't get the drug, but I KNOW he won't if we stay here and wait. Why would they use a placebo if the safety study is done, can't they just do a natural course study of duchenne?
i have heard the study will be in the US, in ohio, but late in 2010, im just afraid austin wont still be walking, so i want him in now! its funny, we thought for years that my boys had a deletion of 51, i even joined the group on PPMD but we recently found out it is 52.i believe the next drug they are beginning trials for in exon skipping will help those with a 51 deletion.


Julie Hathaway said:
My son will be 7 in July. He has a deletion on exon 51. I don't think he would benefit from this trial. Am I correct? I just returned from the conference and the news about exon skipping is very exciting. Kathy Busby from the UK stated that trials would be taking place in the U.S. I think my son needs 50 or 52 skipped. Blessings to all of you.

jenn said:
I actually have a cousin living in london for another year or so...if I hear anything from the trial folks I can provide them a local address,of course we'd rent once we got there, but at least that's a start. Yes, I think we should make our son's known as candidates for trials, older boys won't have a chance by the time trials hit the us. I don't know how I feel about the chance that austin won't get the drug, but I KNOW he won't if we stay here and wait. Why would they use a placebo if the safety study is done, can't they just do a natural course study of duchenne?
We all want the best for our children with DMD. We are all very anxious and involved. We are not trying to bombard anyone with misinformation. But we do get different information from different sources. My information sources come from CNMC in DC and Dr. Eric Hoffman. This is a site where people can come not only for hand holding, but for information. Information one can take and ask their doctors about.

Mindy said:
Hello all,
I think we all need to be careful about a couple of things:
1. These trials are very preliminary right now. The clinic has not met with the FDA to establish protocol, final qualifications, etc. I don't think we should speculate on any of that information until it is finalized so that we don't spread rumors. Lindsay has said that she'll be in touch with the families personally with any developments, so if you hear anything from anyone other than her, don't take it as the final word.
2. We need to be respectful of the people planning these trials, including being careful about posting emails from them, etc. without their permission. Trials like these are very fragile in the planning stages, and agitating in any way doesn't help the process.

IHere are the facts of the trial from Lindsay:
it is systemic, not intramuscular
we don't know dosage
there are risks involved in participating and no guarantees

'm not trying to be the cop here, but I don't want Mendell's office bombarded with controversy and conflict when they're just trying to assess interest in participation.

Thanks,
Mindy
Hey Jenn,

My son has a deletion of 52 and I am desperately trying to find someone trying 52. Columbus is doing 51 at present...if you hear, please let me know. thanks

JoAnne

jenn said:
i have looked at the web site, but it doesn't say anywhere that there is a trial in columbus, only london... also is this just a safety trial or will it actually produce results( hopefully)? i sent our info, i have 2 boys, we had thought they had a deletion of 51, but it is 52, so i think they qualify for this.
Hi JoAnne,

With a deletion of exon 52 your son needs exon 51 skipped which is the first one they (AVI Biopharma and Prosensa) are working on.

Plase take a look here:
http://www.avibio.com/duchenne_muscular_dystrophy.php

Ofelia
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JoAnne Hathcoat said:
Hey Jenn,

My son has a deletion of 52 and I am desperately trying to find someone trying 52. Columbus is doing 51 at present...if you hear, please let me know. thanks

JoAnne

jenn said:
i have looked at the web site, but it doesn't say anywhere that there is a trial in columbus, only london... also is this just a safety trial or will it actually produce results( hopefully)? i sent our info, i have 2 boys, we had thought they had a deletion of 51, but it is 52, so i think they qualify for this.

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