Exon 51 skip (GSK2402968) in ambulant boys Canada

Phase III GSK exon 51 skip  ambulatory boys, recruiting.
48 week trial, Montreal, we are starting soon.

weekly injection (subcutaneous), 2 muscle biopsies, etc,etc.

 

Here we go!

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Just a little update,

Simon is having his 8th injections tomorrow, and he has been having some minor, local skin reactions around some of the injection sites, redness and a bit puffy but not itchy, and anywhere from 1.5 cm to 4.5 cm. Twice on his tummy, once on his arm and once on his leg. It seems to be like eczema. They thought it could be from the alcohol disinfectant, but last week they used chlorohexidine instead and he reacted anyway. No one seems too concerned, just curious, and they prescribed a hydrating cream.

He is the first one that will be getting a muscle biopsy (week 8) on Nov.28th, on the upper calf area by incision. This first biopsy is chosen at random by computer as week 8, 12, 24 or 36). The second biopsy will be at week 48 for all the boys.

I believe that the 7th participant will begin injections tomorrow also if he passes all the selection tests (including 6 minute walk test). So that means the trial will be finished the end of October 2012, plus a few months to analyse all the data. Hopefully we will have some answers by early 2013.

A.

Dear Andrea,

Thanks for sharing. Heartfelt pray for Simon and the boys.

Trinh

Andrea Cleary said:

Just a little update,

Simon is having his 8th injections tomorrow, and he has been having some minor, local skin reactions around some of the injection sites, redness and a bit puffy but not itchy, and anywhere from 1.5 cm to 4.5 cm. Twice on his tummy, once on his arm and once on his leg. It seems to be like eczema. They thought it could be from the alcohol disinfectant, but last week they used chlorohexidine instead and he reacted anyway. No one seems too concerned, just curious, and they prescribed a hydrating cream.

He is the first one that will be getting a muscle biopsy (week 8) on Nov.28th, on the upper calf area by incision. This first biopsy is chosen at random by computer as week 8, 12, 24 or 36). The second biopsy will be at week 48 for all the boys.

I believe that the 7th participant will begin injections tomorrow also if he passes all the selection tests (including 6 minute walk test). So that means the trial will be finished the end of October 2012, plus a few months to analyse all the data. Hopefully we will have some answers by early 2013.

A.

Thank you for your son's participation Andrea.  I'm certain this isn't easy (Patrick participated in the pharmacokennetics arm at Nationwide Children's last year and had skin discoloration at the injection site.).  Between travel, rearranging schedules and  not knowing what affect this is having makes it stressful.  This is a lot to ask from anyone, yet absolutely necessary and I am grateful that families like yours are willing to undergo the challenge.

Brian

Hi Andrea,

We are very much grateful to you and your son and other boys participating. thanks for sharing.

Amrit

 

Thank you so much for sharing this info with us..keep on with faith!!

 

JP.

I just realized that I never posted here this presentation I was able to find. I looked very hard for some data but could not find it until this week. Here are some results for the 48-week extension trial in the first boys who participated in the Prosensa/GSK skipping 51 trial. Individual data on slides 14-15. Looks promising to me. The only boys not improving in the 6MWT are those defined as "in decline" (not sure exacltly how that is defined). Since 6MWT is such a sensitive measure I would not expect to see increase in boys close to the wheelchair stage, that does not mean that other upper body functions do not improve, that data is not presented here. One ca see improvement in 6MWT at weeks 12, 24, 48 in the other boys. By now they have 96-week of data collected. It would be interesting to see what happened in the 2nd year of dosing in these 10 boys.

Attachments:

How is Simon holding up with all of the injections? 

Some of the injections seem to hurt a bit, and sometimes he has a bruise along with the skin reaction, but he is a little trouper, as all the boys are. Some of the other participants find it harder to get the injections, but they offer numbing cream if they wish, which seems to help.

The passing protein in the urine has begun, so they are keeping a close watch on that and we may need to repeat a 24 hour urine collection soon. The study physician added a kidney ultrasound and calcium and vitamin D blood levels to all of the boys, as some are producing crystals (but I was told some were even before the injections began). The nurses did not have the report back yet last week, but I should find out this Thursday.

 

Thank you for asking,

Andrea

Thank you Andrea.. every single one of us have you and Simon in our prayers::thank you for posting.

Andrea Cleary said:

Some of the injections seem to hurt a bit, and sometimes he has a bruise along with the skin reaction, but he is a little trouper, as all the boys are. Some of the other participants find it harder to get the injections, but they offer numbing cream if they wish, which seems to help.

The passing protein in the urine has begun, so they are keeping a close watch on that and we may need to repeat a 24 hour urine collection soon. The study physician added a kidney ultrasound and calcium and vitamin D blood levels to all of the boys, as some are producing crystals (but I was told some were even before the injections began). The nurses did not have the report back yet last week, but I should find out this Thursday.

 

Thank you for asking,

Andrea

Andrea - it sounds as though your son is definitely receiving the medication rather than the placebo!

Hi Andrea,

Can you describe the skin reaction that your son has with injection? Does he have it every time?
Thanks.

Hello Mindy,

Yes he has had the reaction every time, but it is always much less when in the thigh for some reason, with the tummy being the worst to react.  The reactions never appear the same day or evening, only by the next morning. I will try to post a picture that I took of his arm (we were asked to do the outline with a marker to check if it gets bigger or not, which it does not. You will notice that I was not very accurate for the lower site, early in the morning before my coffee).  There will be a well defined area of pinkness and it will be slightly raised (evenly puffed up) about 1 or 2mm. It is dry and looks like eczema, but not itchy (the first time on the tummy it was itchy from being too dry and his waist band rubbing on it, but after about 4 days) , so they prescribed a moisturizer and a mild topical cortisone cream (approved by GSK) which we often forget to even put since they do not really bother him. He is left with a slight pink mark at each injection site, but he is fair skinned and scars easily, so I think he will have the marks until next summer when he tans again (if the spots will tan).

When I read the previous trial report they mentioned skin reactions on the boys, but of course there were no pictures, so I assume this is the type the researchers were describing. The previous trial was also always injected into the tummy, so I assume that is why they have chosen to alternate between the arms, legs and tummy in this trial.

Ta ta for now,

Andrea                 

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