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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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Permalink Reply by Andrea Cleary on March 18, 2012 at 9:07am

Me too...and Utrophin upregulation...and hope Accelron gets off their tuffet!

I see little improvements, but nothing spectacular, and it took about 10 weeks before we noticed any difference.

Good luck to you.

Tulika said:

Thanks for sharing this. Gives me hope since we are with the real stuff for only 4 weeks.

You are seeing improvements which is a sure winner for the trail as a whole.

Hoping that 45 and 53 trails also start soon, and we have class approval for all kids.

Permalink Reply by Rahul Grover on March 18, 2012 at 11:10pm

Thanks Andrea! This is really wonderful to hear!

Permalink Reply by JoAnne Powell on April 8, 2012 at 1:58pm

Andrea,

We just started my son Landon's exon skipping trials in Columbus, Ohio. He has had 2 injections so far, one he had the skin reaction (in his stomach) but this last one was in his arm and no reaction at all. Did your son have reactions each time or from what I understand from your posts, it looks like some injections did and some didn't??? I guess I am like all of us and just hoping to be on active drug....not to mention all the excitement and nervousness from just being in the trials.

Permalink Reply by Andrea Cleary on April 8, 2012 at 4:02pm

Hi JoAnne,

Most of the time Simon reacts, and always does in the stomach, and it's always worse on the stomach, and the lightest on the thigh. The few times there was no reaction it was on the thigh. He sort of has a permanent red demarcation like an upside down horse-shoe all around his belly button now. It will be interesting to see what happens this summer with some swimming and sun.

We are past the excitement stage now, just past 6 months, and waiting for the end, which means the second biopsy just before starting middle school (grade 7).

Do you have to travel far with Landon for the trial? How is that going?

Good luck to you and say hi to Landon for us.

Andrea

Good luck and

Permalink Reply by Laurie Paschal on April 8, 2012 at 5:43pm

About the same with Sam. Worst reaction (and most painful) in the stomach. But he has more reaction in the thigh than the arms.

We've got 6 treatment visits left and then the biopsy. So ready for a break now.

Andrea Cleary said:

Hi JoAnne,

Most of the time Simon reacts, and always does in the stomach, and it's always worse on the stomach, and the lightest on the thigh. The few times there was no reaction it was on the thigh. He sort of has a permanent red demarcation like an upside down horse-shoe all around his belly button now. It will be interesting to see what happens this summer with some swimming and sun.

We are past the excitement stage now, just past 6 months, and waiting for the end, which means the second biopsy just before starting middle school (grade 7).

Do you have to travel far with Landon for the trial? How is that going?

Good luck to you and say hi to Landon for us.

Andrea

Good luck and

Permalink Reply by Donna Cicardo on March 7, 2013 at 7:52pm

hi I was just wondering if you know anything about crystals in the urine. we are not in study but my son has been getting crystals and mucus in urine, maybe you know why thanks.

Andrea Cleary said:

Simon weighs about 34 kilos. Here there was an issue with boys having crystals (separate issue than the protein) in their urine also (some before ever receiving the exon skipping drug or placebo), so the study physician added a kidney ultrasound and calcium and Vit. D blood tests, which we have done anyway as part of his routine DMD follow-up.

Does Sam take Deflazacort or predni? Good that you will be able to do the extension study from London. I had not thought to ask yet, but will tomorrow when we go. Did you move to the UK solely to participate in the trial, or was it a pleasant 'side effect' of somethng you were planning anyway?

Do you know if we are able to get the results of the muscle biopsies at the end of the 48 weeks? And has anyone said what will happen at the end of the extension study? Sometimes it seems that the parents have more questions than the study staff can answer.

Good luck to you, and perhaps we will meet someday, or the boys will meet.

Andrea

Permalink Reply by Andrea Cleary on March 7, 2013 at 8:43pm

Hi Donna,

Urine crystals are usually a normal occurance and can be made of different chemicals, like calcium, oxalates or uric acid. They can appear or disappear based on things like changing pH (acidity) of the urine (sometimes by what we are eating and drinking or a med we are taking) or concentration of the urine. It's always a good idea to stay well hydrated (water is always best), because crystals may be a precursor for kidney stones if they are not "washed out" by having dilute pee (been there, done that, had stone when pregnant and was taking calcium supplements) Mucus is also normal but may increase with an infection or stone passing, etc because it is a protective substance. We are usually giving our DMD kids lots of calcium, vitamins/minerals and various medications which get broken down and can all interact chemically with each other to precipitate out of solution as crystals. It's not good to delay urinating when we have to go by trying to hold it back, and drinking more causes more trips to the bathroom, which is difficult for our guys sometimes to do, I understand. So don't stop taking vitamins and supplements, but he should drink more water as part of his normal routine.

Andrea

Permalink Reply by Dori on June 22, 2016 at 5:38am