New trial of IGF-1 in Duchenne Muscular Dystrophy at Children's Hospital Medical Center, Cincinnati

Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy
This study is not yet open for participant recruitment.
Verified by Children's Hospital Medical Center, Cincinnati, September 2010
First Received: September 22, 2010   No Changes Posted
Sponsor: Children's Hospital Medical Center, Cincinnati
Collaborators: Tercica- Subsdiary of Ipsen
Charley's Fund
Information provided by: Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier: NCT01207908

IGF-1 stimulates both the proliferation and differentiation of skeletal muscle cells and is thus important for muscle repair and regeneration. IGF-1 offers potential as a therapeutic agent for DMD as it may improve or preserve motor function and reduce GC side effects such as growth failure and insulin resistance.

http://www.clinicaltrials.gov/ct2/show/NCT01207908?term=duchenne+mu...

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What is IGF-1? I am not sure I heard this before.
Good!
Cheers for this one.
i have never heard of this either
Isn't this stimulated by growth hormone? I'm assuming it is some type of GH therapy they use in some boys?

http://www.tercica.com/content.php?content_id=17
oh that makes sense, flannagan sort of touched on growth hormone treatment in cincinatti, also have heard there would be a clinical trial soon. now to decide which trial i try to get Max in, this one, or the illusive AVI trial scheduled to happen in columbus later this fall....he cant do both

Ofelia Marin said:
Isn't this stimulated by growth hormone? I'm assuming it is some type of GH therapy they use in some boys?

http://www.tercica.com/content.php?content_id=17
It's great to have options,but with these options come a HUGE responsiblity...basically our sons lives and well-being!!
I too was struggling between AVI {according to the Columbus people, it's supposed to be approved by the end of this year} and Prosena {which Dr. Wong told me in July, Prosena was redoing the tests--12wks instead of the 8 wks which was good enough for the entire European community. By MY math these tests should be ready to present to the FDA in Oct/Nov.....lets hope the FDA approves both!!!!!} Now this new IGF-1....my son falls into the exon 51 category,thank God he's still completely ambulatory, but he'll turn 11 in Jan. Because of his age, I feel I have one shot at a trail.....and I HAVE to get it right!!!! Any info would be appreciated.....I know that all this clinical trial stuff is a personal choice, but I've always felt the more informed a person is the better their choices are!!..........Thanks!!!!
v

jenn said:
oh that makes sense, flannagan sort of touched on growth hormone treatment in cincinatti, also have heard there would be a clinical trial soon. now to decide which trial i try to get Max in, this one, or the illusive AVI trial scheduled to happen in columbus later this fall....he cant do both

Ofelia Marin said:
Isn't this stimulated by growth hormone? I'm assuming it is some type of GH therapy they use in some boys?

http://www.tercica.com/content.php?content_id=17
Karen, it is very difficult to know what to do w/o clear deadlines. The AVI trial in Columbus (planned to start before the end of the year) is a small 12-week trial at high doses. If the results are good it will be followed by a longer term trial but no one knows if or when that's going to be. GSK trial is longer term but the dose is lower and people seem to think that the chemistry used by GSK/Prosensa is not as "effective" as the one used by AVI. Obviously, no one will know if that will be the case in humans until these trials are completed...

There there is ACE-031 supposed to start next year if the Canadian trial goes well I assume. And this IGF-1.

No matter how we look at this, it's great to see progress and options, however this doesn't make your decision easier Karen...

Karen flor said:
It's great to have options,but with these options come a HUGE responsiblity...basically our sons lives and well-being!!
I too was struggling between AVI {according to the Columbus people, it's supposed to be approved by the end of this year} and Prosena {which Dr. Wong told me in July, Prosena was redoing the tests--12wks instead of the 8 wks which was good enough for the entire European community. By MY math these tests should be ready to present to the FDA in Oct/Nov.....lets hope the FDA approves both!!!!!} Now this new IGF-1....my son falls into the exon 51 category,thank God he's still completely ambulatory, but he'll turn 11 in Jan. Because of his age, I feel I have one shot at a trail.....and I HAVE to get it right!!!! Any info would be appreciated.....I know that all this clinical trial stuff is a personal choice, but I've always felt the more informed a person is the better their choices are!!..........Thanks!!!!
v

jenn said:
oh that makes sense, flannagan sort of touched on growth hormone treatment in cincinatti, also have heard there would be a clinical trial soon. now to decide which trial i try to get Max in, this one, or the illusive AVI trial scheduled to happen in columbus later this fall....he cant do both

Ofelia Marin said:
Isn't this stimulated by growth hormone? I'm assuming it is some type of GH therapy they use in some boys?

http://www.tercica.com/content.php?content_id=17
the trial in canada, wont, according to the responses i have gotten, be accepting U.S boys , so that takes it off the list for me. as for prosensa or AVI i think we will do whichever comes first for max, max is 8, still walking, and i really want to keep it that way. austin did the prosensa trial non ambulatory and we hope to get good news by january that they will be doing an extension study. HOPE being the key word. it is so good to see so many things coming, but they need to hurry!

Ofelia Marin said:
Karen, it is very difficult to know what to do w/o clear deadlines. The AVI trial in Columbus (planned to start before the end of the year) is a small 12-week trial at high doses. If the results are good it will be followed by a longer term trial but no one knows if or when that's going to be. GSK trial is longer term but the dose is lower and people seem to think that the chemistry used by GSK/Prosensa is not as "effective" as the one used by AVI. Obviously, no one will know if that will be the case in humans until these trials are completed...

There there is ACE-031 supposed to start next year if the Canadian trial goes well I assume. And this IGF-1.

No matter how we look at this, it's great to see progress and options, however this doesn't make your decision easier Karen...

Karen flor said:
It's great to have options,but with these options come a HUGE responsiblity...basically our sons lives and well-being!!
I too was struggling between AVI {according to the Columbus people, it's supposed to be approved by the end of this year} and Prosena {which Dr. Wong told me in July, Prosena was redoing the tests--12wks instead of the 8 wks which was good enough for the entire European community. By MY math these tests should be ready to present to the FDA in Oct/Nov.....lets hope the FDA approves both!!!!!} Now this new IGF-1....my son falls into the exon 51 category,thank God he's still completely ambulatory, but he'll turn 11 in Jan. Because of his age, I feel I have one shot at a trail.....and I HAVE to get it right!!!! Any info would be appreciated.....I know that all this clinical trial stuff is a personal choice, but I've always felt the more informed a person is the better their choices are!!..........Thanks!!!!
v

jenn said:
oh that makes sense, flannagan sort of touched on growth hormone treatment in cincinatti, also have heard there would be a clinical trial soon. now to decide which trial i try to get Max in, this one, or the illusive AVI trial scheduled to happen in columbus later this fall....he cant do both

Ofelia Marin said:
Isn't this stimulated by growth hormone? I'm assuming it is some type of GH therapy they use in some boys?

http://www.tercica.com/content.php?content_id=17
I agree Jenn! The clock is ticking!

jenn said:
the trial in canada, wont, according to the responses i have gotten, be accepting U.S boys , so that takes it off the list for me. as for prosensa or AVI i think we will do whichever comes first for max, max is 8, still walking, and i really want to keep it that way. austin did the prosensa trial non ambulatory and we hope to get good news by january that they will be doing an extension study. HOPE being the key word. it is so good to see so many things coming, but they need to hurry!

Ofelia Marin said:
Karen, it is very difficult to know what to do w/o clear deadlines. The AVI trial in Columbus (planned to start before the end of the year) is a small 12-week trial at high doses. If the results are good it will be followed by a longer term trial but no one knows if or when that's going to be. GSK trial is longer term but the dose is lower and people seem to think that the chemistry used by GSK/Prosensa is not as "effective" as the one used by AVI. Obviously, no one will know if that will be the case in humans until these trials are completed...

There there is ACE-031 supposed to start next year if the Canadian trial goes well I assume. And this IGF-1.

No matter how we look at this, it's great to see progress and options, however this doesn't make your decision easier Karen...

Karen flor said:
It's great to have options,but with these options come a HUGE responsiblity...basically our sons lives and well-being!!
I too was struggling between AVI {according to the Columbus people, it's supposed to be approved by the end of this year} and Prosena {which Dr. Wong told me in July, Prosena was redoing the tests--12wks instead of the 8 wks which was good enough for the entire European community. By MY math these tests should be ready to present to the FDA in Oct/Nov.....lets hope the FDA approves both!!!!!} Now this new IGF-1....my son falls into the exon 51 category,thank God he's still completely ambulatory, but he'll turn 11 in Jan. Because of his age, I feel I have one shot at a trail.....and I HAVE to get it right!!!! Any info would be appreciated.....I know that all this clinical trial stuff is a personal choice, but I've always felt the more informed a person is the better their choices are!!..........Thanks!!!!
v

jenn said:
oh that makes sense, flannagan sort of touched on growth hormone treatment in cincinatti, also have heard there would be a clinical trial soon. now to decide which trial i try to get Max in, this one, or the illusive AVI trial scheduled to happen in columbus later this fall....he cant do both

Ofelia Marin said:
Isn't this stimulated by growth hormone? I'm assuming it is some type of GH therapy they use in some boys?

http://www.tercica.com/content.php?content_id=17
IGF-1 is already FDA approved, so you know that whats going into your child is not unknown. It will be injected by you into your son daily (i believe daily). You have to make 3 trips to Cincy, begining, middle, and end. Recruiting starts in October and they hope to start by March 2011. One side effect they will monitor is the deterioration of the fluid between the brain and skull as, for lack of a better word, can dry when taking IGF-1.

For the exon skipping trials, does anyone know the amount that will be used during the studies? Will it be enough to be beneficial for the entire body or will they be focused on one area (e.g. the foot)?
Both AVI and GSK exon skipping trials are systemic delivery to the whole body, however we do not know at this time how effective it will be in certain muscle groups. The dose used in the AVI trial will be quite high, 50 and 100 mg/kg (as opposed to 20 mg/kg that was used in the UK trial that produced mixed results, dystrophin in all subjects but variable % from patient to patient with only one showing 55% dystrophin positive fibers). The do believe that the dose in the UK trial was too low hence decide to increase it a lot in the planned US trial. GSK is using a lower dose level from what I know, but it's a longer trial 48 weeks? AVI is only 12 weekly injections, the next phase should be 48 weeks or so, if this short trial gives positive results.



MicahsDaddy said:
IGF-1 is already FDA approved, so you know that whats going into your child is not unknown. It will be injected by you into your son daily (i believe daily). You have to make 3 trips to Cincy, begining, middle, and end. Recruiting starts in October and they hope to start by March 2011. One side effect they will monitor is the deterioration of the fluid between the brain and skull as, for lack of a better word, can dry when taking IGF-1.

For the exon skipping trials, does anyone know the amount that will be used during the studies? Will it be enough to be beneficial for the entire body or will they be focused on one area (e.g. the foot)?

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