PTC 124-2b Clinical Trial


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


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 brad stringer on November 14, 2008 at 1:37am
Hi Neeraj,

I understand your perspective that you want to do all you can for Aran. how is he doing right now, and did he have his first biopsy yet?
Comment by neeraj on October 22, 2008 at 1:44pm
hi Brad

We went to the day one of the screening yesterday with aran(8.4) year old.The day was very intense and this is just a start.So i accept the next months i will be spending all my days off at the hospital but i think more for aran what he has to go through but also reading about ptc124 it gives me confidence that it has the ability to treat aran hence our answer to any hope to save my son.Take the other side of the coin doing nothing is not going to do anything and i want to take every oppurtunity to save my son so even a lot of pain for 12 or so months will still be lot less than what future illness holds.I just know i have to get aran in this trial to let him have a chance.
Comment by brad stringer on October 22, 2008 at 12:01am
Sorry, I think the correct number is 38 boys in the 2a study(?)
Comment by brad stringer on October 21, 2008 at 11:57pm
Last year 26 boys participated in the Phase 2a PTC124 trial, and now 165 boys will embark on phase 2b (or they have already). Many families have made the difficult decision to enter into the trials and their extensions, and many more are now in the middle of this decision making process or will be soon.

At this point personally, I am trying to gather as much information about the trial as possible so that I can make the best decision for my son and our family. To be honest, I am really struggling. When I first learned that our son was eligible I was elated, but now after reading the consent form and some personal, beautifully written accounts from the 2a extension discussion group, I am not quite sure it is all worth it for our son. Certainly for the greater good and the push towards a final cure for all DMD kids, it is absolutely worth it.

Nevertheless, my little guy is only 5 years old. Considering the specified regimen of regular 2 day clinic visits every 6 weeks, the high number of blood draws and the necessity of two biopsies over the course of 10 months plus the potential for more of everything in the following 2 years extension period, I am really struggling to decide whether the benefits outweigh the drawbacks.

Since one of the keys to participating in this trial is immediate qualification into the extension study, the full study could run approximately 3 years.
The extension is key for the 2b study because all participants will receive the study drug there since there is a 1/3 chance of a placebo in the 10 month trial.
Overall, it’s a lot of hospital time, a lot of Clinic Time and a lot of blood work for a little child.
I guess my main concerns as I near a decision are the following:

1-What was the true measurable increase in dystrophin production from the 2a study? -I have not seen any actual numbers in any of the published materials to date
2-What is the potential for serious side effects to develop over a 10 month period? -28 days for 2a was very short and not really a true indicator of long term safety.

3-Is the 3 years of a hospital-centered lifestyle really worth it for our son? -If we agree to participate, then Skylers childhood memories from the age of 6 to 9 will be dominated by the memories of his time in hospital, out of school lots, and being tested, poked, prodded, attached to a motion sensor, operated on and forced to take medicine 3 times a day.
Quite frankly, in the absence of clear proof of a real benefit, I am not so sure subjecting him to all of this is worth the sacrifice to the quality of his still carefree young childhood days.

I know many of you have struggled with and resolved these issues in your own families, and many are now also looking for any suggestions to help make such an important decision in the lives of our sons.

Any input would be greatly appreciated.

Cc; “ 2a Study Families”
Comment by brad stringer on October 17, 2008 at 2:38am
**This is a new group for any current PTC 124 Clinical Trial 2a or 2b participants or those considering entering this trial.

Briefly, PTC 124 is an experimental drug under investigation for boys (or girls) with a Premature Stop Codon/ Nonsense mutation. If you are not sure if your child may be eligible, you will need to have the mutation confirmed by dystrophin gene sequencing. Once confirmed, your neuromuscular clinic and medical staff will further advise on particulars of the Clinical Trial itself.

For more information go to:

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