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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Hi Andrea,

My son Sam is in the trial in Lille, France. We live north of London in the UK but are originally from Texas. This Thursday will be his 28th injection. Sam gets the same red spots with his tummy also being the most reactive. Also gets some bruising. Have they ever given your son two injections on the same day? My husband took Sam last week and he received two injections. He didn't think to ask why until Sam asked him about it after they left. How did his biopsy go? Sam's site was very slow to heal, even opened back up after they took the stitches out.

Hope all is still going well for y'all.

Laurie

How much does Simon weigh? Sam wavers between 28 and 29kgs. I don't know if we've seen much effects. I think Sam was about to start to decline before starting the trial. He does have lots of energy. He's had a few side effects besides the skin reactions. He's had protein once that I know of and also had low coagulation numbers once.

The travelling has gotten old. Never thought I'd say I was tired of going to France. We leave the house at 4am. Take a train to London, then the Eurostar train to Lille. We get home between 3:30 and 4:30 depending on how on schedule the Eurostar is in the afternoon.

We decided to home-school all the kids during the trial. It's just easier to not have to worry about missing and make up work. My daughter is 13 and watches Sam's twin brother Jake while we're gone during the day. Luckily we found out that Sam will be doing the extension trial out of London. We plan on enrolling him and the others in the local schools in the Fall. Hopefully it won't interrupt things too much.

I would love to be able to touch base with others. I know Mindy who's son is participating in Vancouver. Also know a few who are in the phase 2 here in the UK.

Laurie

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

Sam is on Deflazacort. We moved here with the intention of enrolling him in the trial.It was 100% guaranteed he'd get in, but we had to try.

I think the biopsy results are only available after the entire trial is over, or at least when all boys at your center are finished. I haven't asked, but have spoken with some others. I don't know what happens at the end of the extension either. I have such a hard time asking questions b/c I know very little French and the doctors/nurses are only marginally better in English

I'd love to be able to meet. We've talked about moving to Canada if the US still isn't on board when we want/need to move from here.

Laurie

All our faith with you!

Thanks for the support Juan!

Thanks from us too! Yours and your son's bravery means a lot to so many people. You're pioneers of a better future.

Just got word today that the extension study will be 107 weeks! We are at week 17 today, 31 more to go + 107 = Holy Moly!

Wow, almost three years in the study.  Certainly ought to know how well it's working for Simon by then.  My prayers remain with you.

Andrea Cleary said:

Just got word today that the extension study will be 107 weeks! We are at week 17 today, 31 more to go + 107 = Holy Moly!

Their timeline is here: https://www.duchenneconnect.org/index.php?option=com_content&vi...

Which also shows that this drug cannot be approved earlier than 2015! I think that we can all do the math on how long it will take for the drugs not even in clinical trial now, other exons, urtrophin upreg, best case scenario 5+ years away.



Andrea Cleary said:

Just got word today that the extension study will be 107 weeks! We are at week 17 today, 31 more to go + 107 = Holy Moly!

Half way through at week 24.

Doing well, same skin reactions at the injection site, and apparently he is the one who reacts the most (with sensitive skin). Where they inject into the deltoid (upper arm) the reaction site has become a bit hard, like a little mound of denser flesh. May just be fibrosis due to progression of DMD, time will tell.

I find his calf muscles softer, less hard and rubbery, but no improvement in Achilles stiffness at the ankles. Quadriceps (front of thigh) seem to have shrunken, more on the right than on the left, which has always been bigger. He has lost 3 kg in the 6 months since we started this trial, without much explanation. We have been 'careful' about calorie intake, but not obsessively so, and only since December.

Can skip (run while hopping, not skipping rope) for the first time in his life. Runs a bit better (less chest/tummy stuck out), straighter and faster, but only for short distances. Can jump a bit better and higher.

It just seems like he has more energy, talks more, etc, but maybe that is just age, maturity and confidence. He can play a game of basketball for about 20 minutes at Scouts before having to stop. I think he is doing better on stairs. Have not had any major falls lately (since early December 2011).

Passing protein in the urine, but it is not causing problems at this point. Underwent a sleep study (not in relation to the trial) because he snores, but there were no periods of apnea recorded.

 

 

 

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.

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