PTC 124-2b Clinical Trial

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PTC 124-2b Clinical Trial

Various issues re: PTC 124 Clinical Trials for current or prospective participants worldwide

Members: 31
Latest Activity: May 1, 2018

Discussions

PTC124 now has a name...

Started by Paul Johnson. Last reply by Paul Johnson Feb 3, 2009. 1 Reply

PTC124 in the courts

Started by Paul Johnson. Last reply by Paul Johnson Dec 22, 2008. 4 Replies

Is the 2b Trial Full?

Started by Paul Johnson. Last reply by brad stringer Dec 4, 2008. 1 Reply

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Comment by Paul Johnson on November 15, 2008 at 11:19pm
Hey Brad...
Elliott (thankfully...) had always been fairly good so far at the Dr's, with Blood draws etc... so we've been blessed with that. However, knowing Weds, was going to be a long day, we had a present wrapped up and sitting next to him in the car. We told him if he was a good listener throughout the day that he could have the present when the day was done. Oddly - he didn't ask us much about the present, but when he was getting a little tired, we borught it up and gave him clues and played a guessing game.
The other key to the day was giving Elliott something to do in those down times - he's stil young and a friend of us gave us a Fisher Price Kid Tough music player figuring it would be helpful. So the night before I loaded up some of his favorites songs and also some audio stories he likes... and that was wonderful for those time where we were waiting - couldn't imagine going down to our Clinic visits without it now.
Comment by brad stringer on November 15, 2008 at 10:42pm
Angela,
Thank you for all of this fantastic information.Its very helpful, as I have no ideas about how to help (Distract/ persuade) Skyler on these difficult days.
The gifts, pinwheel, DS are all great! I am thinking maybe one of those little portable dvd players?
Have you ever tried that?
The pinwheel is also a great idea!
Its interesting that he was exposed to the needle and played with it too. A great idea.
Our Hospital has a Child Life person who can help kids prepare for and feel better with their procedures. I will make sure they are involved, for sure.
By the way...is it possible you can find out from your doctor exactly what the "giggle juice" sedation was?
Skyler needs some sedation in the next week for a dental procedure, and I am having trouble getting full information on acceptable/ unacceptable sedatives for DMD kids.
Only one I know for sure is never to be used is SUCCYNOCHOLINE-Its an inhalation sedative for general, but our dentist wants to use an oral sedative at the start.
Any help would be appreciated.
Comment by brad stringer on November 15, 2008 at 10:19pm
Hi Erica,
Thank you for your post. You guys are very strong. Can I ask...what was the deciding factor in agreeing to participate in the trial?
Also...what were some of the things you did with Mati to help him through the biopsy and still do to help him get through the blood work? What about drinking the powder 3x a day?
Comment by Erica on November 15, 2008 at 6:19pm
Hi Brad,
Making this decision was very hard for us, too. We decided to go ahead and so our son Mati, who just turned 8, has been on the drug/placebo since April.

So far, the worst part has been the biopsy. He was very nervous and the sedative didn’t help much, in my opinion. But we managed. Now, Mati is seeing a psychologist to help him "prepare" for the next biopsy, which is scheduled for January 7. I hope this one will go smoother.
As for the blood work, he got used to it and does not complain anymore (he did the first couple of times).
Best regards,
Erica
Comment by neeraj on November 15, 2008 at 8:55am
Hi Brad

Most difficult for aran was the biopsy day and also he does not like the blood tests.We keep him motivated with gifts and almost anything he wants,although he is a brave child.I was chasing the hospital for months to get him screened once they started.Eventually things got going in october and then moved very quickly and next tuesday some physical tests and then drug(Hope) on wednesday.I must add that there is a possibility of trial running half the time then planned if the benefits are significant.I would defenetly recommend you getting you son screened to have the best chance of a treatment soon.

Regards and very Best
Neeraj
Comment by brad stringer on November 14, 2008 at 11:00pm
Paul,

Sounds like a very big day for you all, and your son. And more to come as well. How did Elliot do throughout the day, and has he had any bloodwork yet?
How did you help him to cooperate and do all that was required of him??
Comment by Paul Johnson on November 14, 2008 at 3:05pm
We went Wednesday down to Children's Hospital of Philadelphia for our Pre-Screening day - it took just about 6 hours... it included Blood tests, kidney & bowel Ultrasounds, EKG and soem PT tests and standard physical. After they get the results form the medical tests we'll hear back and at tha point we'll schedule an Echocardiogram - and if that is accpetable, then we schedule the biopsy...
I'd like to reiterate what Angela said - they repeated several times, that we are have the right to discontinue the study at anytime if it is too much for Elliott... and as Angela said, I'm sure they don't want that... but the option is there if you decide to participate and then find it too much for Skylers...
Also - I could be wrong, but it seemed like to me from the discussion, like the trial is filling up quick and nearing the 165 participants, so the decision may be made for you... ie. no slots left. But ultimately no matter what anyone of us may say pro or con... the decision is yours... and because it is a study, I don't think anyone can offer the golden piece of info to help you detemine if you'd be wrong not to participate or the inverse you'd be wrong if you did participate. Whichever decision you make be certain in the knowledge that you made the right decision for you and your family regardless.
Comment by brad stringer on November 14, 2008 at 9:55am
Angela...wow, you have quite an ordeal with that long travel time thrown into the mix of everything else!I want to ask you the same question as well...what were the toughest things about the biopsy, and how did you handle them with your son to make it easier for him?
Comment by brad stringer on November 14, 2008 at 9:52am
Neeraj,
just wondering...what were the toughest things about the biopsy, and how did you handle them with Aran to make it easier for him?-Brad
Comment by neeraj on November 14, 2008 at 5:09am
Hi Brad,

hope everything is very well and our wish for our boys treatment is soon granted.
Aran is doing well and goes to main stream shool.He had his biopsy on 5th november,It was not an easy day for him but our hope for a treatment is the gain after this pain.We are going on 18-19 november for the drug(Positive hope and prayers). and then get on from there.

So far it has been ok but not easy but as i always hope this things can get better from here on.Lets hope this trial only lasts half the time as there is a possibility that if the results are significant they may unblind the trial in half the time and everyone put on high dose.We remain positive and hope time goes quick in trial.

Regards
Neeraj
 

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