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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Michelle,
What have you heard on Dr. Mendell's trial? I haven't heard anything in forever and am anxious to hear something. The exon skipping will not work for Justin. After rereading the opening post you are referring to the exon skipping trial...thought you were talking about the biostrophin trial.

Kristi

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.
I was told from Laurence Viollet, who is the Certified Clinical Research Coordinator and Team Project Leader for the Center of Gene Therapy:

"We anticipate to start the trial late February, early March of 2010. Of course, we will not enroll all the patients at the same time, so we will try to accommodate everyone’s schedule, depending on availability and if the patient meets all the requirements after the screening visit.
I will contact you back as soon as we have the study approved and we can share the consent form with patients and families."

AND THEN AFTER MY QUESTION OF WHEN AND WHERE

"The study will be done at Nationwide Children's Hospital in Columbus, OH only. That is the only site chosen by AVI for the moment. If AVI decides to enroll more patients, they may chose other sites, but for now, the 32 patients will be seen at Nationwide Children's only.
The clinical protocol needs to be finalized, but basically, there will be weekly injections for 12 weeks, then 5 or so follow-up visits, about 2 or 3 weeks apart.
All the medical expenses will be covered. We will do our best to accomodate hotel rooms through our Welcome Center and to reimburse for travel expenses but that will depend on the money we get from the sponsor. Again, that has to be defined and we are working on it. We will know more in a few weeks.
I will contact you when we have a protocol and a consent form we can share with patients."

My hope and prayers are that this exon skipping work for Nicolas as well as the rest of our boys as a treatment, since we know it's not a cure. And if it doesn't work for my son in his lifetime, then I want Nicolas to make a difference for the future generations, and hopefully end this terrible disease.

Kristi Koop said:
Michelle,
What have you heard on Dr. Mendell's trial? I haven't heard anything in forever and am anxious to hear something. The exon skipping will not work for Justin.

Kristi

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.
Hello All,

I know that many of you had added me as a friend, and I wanted to apologize, for just getting the time to come on and share all the excitement. As many of you know by now in July, I left Nationwide Children's Hospital, as I was offered a position within a private practice to lead a research group! The 5 years I spent at childrens were very precious to my heart, as I got to see many developments like this one, and meet many amazing children and families like yourselves. Dr. Mendell and his group are absolutely AMAZING!!! I couldn't have worked with a better family of people! They pour their hearts and lives into studing to find the cure for Muscular Dystrophy. I truly believe that this team will acomplish just that! I wanted everyone to know, that all our communications were passed on to my amazing collogues before I left. Congratulations! This study is a HUGE advancement, and I am excited for each and everyone of you! God Bless you all and Happy Holidays! We recieved our first "snow" fall in columbus today! Hopefully by March it will be warming up for you out of staters again!!! :)

Best Regards,

Lindsay
Quick question regarding the Ohio Trial - you say "Injections" are you sure you don't mean IV delivery? The point is to raise the dose and look to ensure safely. Injections have already been done. You cannot inject the entire body - I thought this trial was for IV delivery so the dose could be raised. I believe it is - can you clarify this for me, please?? Thanks a bunch.

Michelle Gonzales said:
I was told from Laurence Viollet, who is the Certified Clinical Research Coordinator and Team Project Leader for the Center of Gene Therapy:

"We anticipate to start the trial late February, early March of 2010. Of course, we will not enroll all the patients at the same time, so we will try to accommodate everyone’s schedule, depending on availability and if the patient meets all the requirements after the screening visit.
I will contact you back as soon as we have the study approved and we can share the consent form with patients and families."

AND THEN AFTER MY QUESTION OF WHEN AND WHERE

"The study will be done at Nationwide Children's Hospital in Columbus, OH only. That is the only site chosen by AVI for the moment. If AVI decides to enroll more patients, they may chose other sites, but for now, the 32 patients will be seen at Nationwide Children's only.
The clinical protocol needs to be finalized, but basically, there will be weekly injections for 12 weeks, then 5 or so follow-up visits, about 2 or 3 weeks apart.
All the medical expenses will be covered. We will do our best to accomodate hotel rooms through our Welcome Center and to reimburse for travel expenses but that will depend on the money we get from the sponsor. Again, that has to be defined and we are working on it. We will know more in a few weeks.
I will contact you when we have a protocol and a consent form we can share with patients."

My hope and prayers are that this exon skipping work for Nicolas as well as the rest of our boys as a treatment, since we know it's not a cure. And if it doesn't work for my son in his lifetime, then I want Nicolas to make a difference for the future generations, and hopefully end this terrible disease.

Kristi Koop said:
Michelle,
What have you heard on Dr. Mendell's trial? I haven't heard anything in forever and am anxious to hear something. The exon skipping will not work for Justin.

Kristi

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.
A Prosensa Trial in Ohio? What news do you have on this? I am surprised that two trials would go on during the same year in the same state. Can you share any more news??? Thanks

irishgirl said:
Quick question regarding the Ohio Trial - you say "Injections" are you sure you don't mean IV delivery? The point is to raise the dose and look to ensure safely. Injections have already been done. You cannot inject the entire body - I thought this trial was for IV delivery so the dose could be raised. I believe it is - can you clarify this for me, please?? Thanks a bunch.

Michelle Gonzales said:
I was told from Laurence Viollet, who is the Certified Clinical Research Coordinator and Team Project Leader for the Center of Gene Therapy:

"We anticipate to start the trial late February, early March of 2010. Of course, we will not enroll all the patients at the same time, so we will try to accommodate everyone’s schedule, depending on availability and if the patient meets all the requirements after the screening visit.
I will contact you back as soon as we have the study approved and we can share the consent form with patients and families."

AND THEN AFTER MY QUESTION OF WHEN AND WHERE

"The study will be done at Nationwide Children's Hospital in Columbus, OH only. That is the only site chosen by AVI for the moment. If AVI decides to enroll more patients, they may chose other sites, but for now, the 32 patients will be seen at Nationwide Children's only.
The clinical protocol needs to be finalized, but basically, there will be weekly injections for 12 weeks, then 5 or so follow-up visits, about 2 or 3 weeks apart.
All the medical expenses will be covered. We will do our best to accomodate hotel rooms through our Welcome Center and to reimburse for travel expenses but that will depend on the money we get from the sponsor. Again, that has to be defined and we are working on it. We will know more in a few weeks.
I will contact you when we have a protocol and a consent form we can share with patients."

My hope and prayers are that this exon skipping work for Nicolas as well as the rest of our boys as a treatment, since we know it's not a cure. And if it doesn't work for my son in his lifetime, then I want Nicolas to make a difference for the future generations, and hopefully end this terrible disease.

Kristi Koop said:
Michelle,
What have you heard on Dr. Mendell's trial? I haven't heard anything in forever and am anxious to hear something. The exon skipping will not work for Justin.

Kristi

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.
This just in from Laurence - Ohio trial coordinator:
The purpose of this trial is also to compare IV and sub-cutaneous injections, so we will have 2 groups of patients.
All participants will need to be here one full day every week for 12 weeks. Some visits may require to stay overnight.
I thought we were long beyond the straight injections for efficacy - that goodness we are!!!
Michelle:
WIll there really be a chance that travel expenses will be covered by the trail at least in some way???
That would be totally excellent!
Noreen

Michelle Gonzales said:
I was told from Laurence Viollet, who is the Certified Clinical Research Coordinator and Team Project Leader for the Center of Gene Therapy:

"We anticipate to start the trial late February, early March of 2010. Of course, we will not enroll all the patients at the same time, so we will try to accommodate everyone’s schedule, depending on availability and if the patient meets all the requirements after the screening visit.
I will contact you back as soon as we have the study approved and we can share the consent form with patients and families."

AND THEN AFTER MY QUESTION OF WHEN AND WHERE

"The study will be done at Nationwide Children's Hospital in Columbus, OH only. That is the only site chosen by AVI for the moment. If AVI decides to enroll more patients, they may chose other sites, but for now, the 32 patients will be seen at Nationwide Children's only.
The clinical protocol needs to be finalized, but basically, there will be weekly injections for 12 weeks, then 5 or so follow-up visits, about 2 or 3 weeks apart.
All the medical expenses will be covered. We will do our best to accomodate hotel rooms through our Welcome Center and to reimburse for travel expenses but that will depend on the money we get from the sponsor. Again, that has to be defined and we are working on it. We will know more in a few weeks.
I will contact you when we have a protocol and a consent form we can share with patients."

My hope and prayers are that this exon skipping work for Nicolas as well as the rest of our boys as a treatment, since we know it's not a cure. And if it doesn't work for my son in his lifetime, then I want Nicolas to make a difference for the future generations, and hopefully end this terrible disease.

Kristi Koop said:
Michelle,
What have you heard on Dr. Mendell's trial? I haven't heard anything in forever and am anxious to hear something. The exon skipping will not work for Justin.

Kristi

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.
I was told by Laurence that they will be looking into coverage for travel expenses and help with hotels. I'm sure they'll try and cover what they can, but as Laurence stated, it's up to the company (AVI) as to what exactly will be covered.

Also for the injections, that is what Laurence emailed to me, but in the on-line Quest it did state subcutaneous injections as well as IV.

irishgirl said:
Michelle:
WIll there really be a chance that travel expenses will be covered by the trail at least in some way???
That would be totally excellent!
Noreen

Michelle Gonzales said:
I was told from Laurence Viollet, who is the Certified Clinical Research Coordinator and Team Project Leader for the Center of Gene Therapy:

"We anticipate to start the trial late February, early March of 2010. Of course, we will not enroll all the patients at the same time, so we will try to accommodate everyone’s schedule, depending on availability and if the patient meets all the requirements after the screening visit.
I will contact you back as soon as we have the study approved and we can share the consent form with patients and families."

AND THEN AFTER MY QUESTION OF WHEN AND WHERE

"The study will be done at Nationwide Children's Hospital in Columbus, OH only. That is the only site chosen by AVI for the moment. If AVI decides to enroll more patients, they may chose other sites, but for now, the 32 patients will be seen at Nationwide Children's only.
The clinical protocol needs to be finalized, but basically, there will be weekly injections for 12 weeks, then 5 or so follow-up visits, about 2 or 3 weeks apart.
All the medical expenses will be covered. We will do our best to accomodate hotel rooms through our Welcome Center and to reimburse for travel expenses but that will depend on the money we get from the sponsor. Again, that has to be defined and we are working on it. We will know more in a few weeks.
I will contact you when we have a protocol and a consent form we can share with patients."

My hope and prayers are that this exon skipping work for Nicolas as well as the rest of our boys as a treatment, since we know it's not a cure. And if it doesn't work for my son in his lifetime, then I want Nicolas to make a difference for the future generations, and hopefully end this terrible disease.

Kristi Koop said:
Michelle,
What have you heard on Dr. Mendell's trial? I haven't heard anything in forever and am anxious to hear something. The exon skipping will not work for Justin.

Kristi

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.
Michelle:
THanks so much!!!!
Yes, the trial is for subcutaneous injections as well as IV - not sole injections - as they already did that with flawless results in the UK.
I appreciate all of your post so much!!!
Noreen

Michelle Gonzales said:
I was told by Laurence that they will be looking into coverage for travel expenses and help with hotels. I'm sure they'll try and cover what they can, but as Laurence stated, it's up to the company (AVI) as to what exactly will be covered.

Also for the injections, that is what Laurence emailed to me, but in the on-line Quest it did state subcutaneous injections as well as IV.

irishgirl said:
Michelle:
WIll there really be a chance that travel expenses will be covered by the trail at least in some way???
That would be totally excellent!
Noreen

Michelle Gonzales said:
I was told from Laurence Viollet, who is the Certified Clinical Research Coordinator and Team Project Leader for the Center of Gene Therapy:

"We anticipate to start the trial late February, early March of 2010. Of course, we will not enroll all the patients at the same time, so we will try to accommodate everyone’s schedule, depending on availability and if the patient meets all the requirements after the screening visit.
I will contact you back as soon as we have the study approved and we can share the consent form with patients and families."

AND THEN AFTER MY QUESTION OF WHEN AND WHERE

"The study will be done at Nationwide Children's Hospital in Columbus, OH only. That is the only site chosen by AVI for the moment. If AVI decides to enroll more patients, they may chose other sites, but for now, the 32 patients will be seen at Nationwide Children's only.
The clinical protocol needs to be finalized, but basically, there will be weekly injections for 12 weeks, then 5 or so follow-up visits, about 2 or 3 weeks apart.
All the medical expenses will be covered. We will do our best to accomodate hotel rooms through our Welcome Center and to reimburse for travel expenses but that will depend on the money we get from the sponsor. Again, that has to be defined and we are working on it. We will know more in a few weeks.
I will contact you when we have a protocol and a consent form we can share with patients."

My hope and prayers are that this exon skipping work for Nicolas as well as the rest of our boys as a treatment, since we know it's not a cure. And if it doesn't work for my son in his lifetime, then I want Nicolas to make a difference for the future generations, and hopefully end this terrible disease.

Kristi Koop said:
Michelle,
What have you heard on Dr. Mendell's trial? I haven't heard anything in forever and am anxious to hear something. The exon skipping will not work for Justin.

Kristi

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.
I think people are confusing intramuscular injections, which were the phase 1 trials where they injected a single muscle to see if it produced dystrophin, from subq injections, which is a systemic administration where injections are given into fatty tissue and distributed throughout the body.

The trial in Ohio is a systemic trial, meaning the drug will go to all muscles of the body (hopefully). There are two ways of administering this drug - IV and subq. Subq is much easier to administer - it can be done at home, whereas an IV infusion is much less practical. AVI is hoping that subq administration has as much efficacy as IV infusion, so it's my understanding that they hope this short-term trial will compare the two and demonstrate that.
Also - Prosensa has not officially announced what centers will be participating in their next round of trials. They have been meeting with centers over the last few months, and I've heard that they're announcing in December where the trial will be located.
This is also my understanding. One additional point. Prosensa/GSK trial is using subcutaneous injections (not IV). AVI UK trial is using IV only; this US Columbus trial will be looking at two groups (one using IV, the other using subcutaneous injections) -- one can hope for comparable results, hence easier administration using subcutaneous for next trial phases.

Mindy said:
I think people are confusing intramuscular injections, which were the phase 1 trials where they injected a single muscle to see if it produced dystrophin, from subq injections, which is a systemic administration where injections are given into fatty tissue and distributed throughout the body.

The trial in Ohio is a systemic trial, meaning the drug will go to all muscles of the body (hopefully). There are two ways of administering this drug - IV and subq. Subq is much easier to administer - it can be done at home, whereas an IV infusion is much less practical. AVI is hoping that subq administration has as much efficacy as IV infusion, so it's my understanding that they hope this short-term trial will compare the two and demonstrate that.

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