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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,
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 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.
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.
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:
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.
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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