PTC124 Phase 2A Extension Study
This discussion is to hopefully stay connected with other families of boys who participated on the Phase 2a (38 boys in three sites), and to communicate with all the DMD community the progress of our boys while on PTC124.
My son Daniel Castro (10 years old) participated in the 28 days clinical trial of PTC124 along with other 37 boys a couple of years ago. Since, we have been patiently wating for the extension study. Thank God as of last week, we got the confirmation from Dr. Finkel's team in Philadelphia that everything is ready to go, and they are asking ALL the families of the boys who participated to request the weeks in which they would prefer to go to start the new study. If you are one of those families and you haven't gotten in contact with your hospital in which you did the original trial, please contact them ASAP to get this going for our boys. Great news is that our boys will be taken PTC124 for almost 2 years. They will need to get muscle byopsies again, but that is an small price to pay for the potential benefit we all expect to get out of it.
I encourage parents and families in general of these boys to use this discussion to talk about anything related to the development of this new study, and to report progress, complications, experiences, syntoms, differences noticed while in treatment etc. I think it would be good not only for those 38 families, but hopefully for ALL the kids with non-sence mutation who are participating on the new 2b study and even for all other DMD kids with other type of mutations since PTC therapeutics is working on a new medicine for ALL the cases of DMD.
God bless you ALL.
Jose, Angie, Daniel and the Castro Gang.
My son Daniel Castro (10 years old) participated in the 28 days clinical trial of PTC124 along with other 37 boys a couple of years ago. Since, we have been patiently wating for the extension study. Thank God as of last week, we got the confirmation from Dr. Finkel's team in Philadelphia that everything is ready to go, and they are asking ALL the families of the boys who participated to request the weeks in which they would prefer to go to start the new study. If you are one of those families and you haven't gotten in contact with your hospital in which you did the original trial, please contact them ASAP to get this going for our boys. Great news is that our boys will be taken PTC124 for almost 2 years. They will need to get muscle byopsies again, but that is an small price to pay for the potential benefit we all expect to get out of it.
I encourage parents and families in general of these boys to use this discussion to talk about anything related to the development of this new study, and to report progress, complications, experiences, syntoms, differences noticed while in treatment etc. I think it would be good not only for those 38 families, but hopefully for ALL the kids with non-sence mutation who are participating on the new 2b study and even for all other DMD kids with other type of mutations since PTC therapeutics is working on a new medicine for ALL the cases of DMD.
God bless you ALL.
Jose, Angie, Daniel and the Castro Gang.
Replies to This Discussion
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Permalink Reply by Paul Cliff on
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Good luck and best wishes to all of you brave pioneers and your sons.
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Permalink Reply by dacas01 on
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Thanks Paul.
Paul Cliff said:Good luck and best wishes to all of you brave pioneers and your sons.
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Permalink Reply by Joanne Wechlser on
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Hi Jose, etc,
We too are waiting for the trial to start in Philadelphia. It's now August 23rd and still waiting. We did get an e-mail from Teesha the other day, but she hasn't responded as to when we will get our initial screening bloodkit in the mail. We're anxiously awaiting some kind of schedule or at least more information. Have you heard anything?
Joanne
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Hi Jose,
It's now August 30th. Any news yet? We started a Caring Bridge Site for Adam to keep family and friends informed.
http://www.caringbridge.org/visit/adamwechsler
Joanne Wechlser said:Hi Jose, etc,
We too are waiting for the trial to start in Philadelphia. It's now August 23rd and still waiting. We did get an e-mail from Teesha the other day, but she hasn't responded as to when we will get our initial screening bloodkit in the mail. We're anxiously awaiting some kind of schedule or at least more information. Have you heard anything?
Joanne
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Hi Joanne: Nothing yet. I guess they are really testing our patience. I emailed them last Thursday and got the regular... we are working on it... There isn't much we can do other than wait. I feel it will be just a matter of a few days before we get this local test done and then schedule our visit to Philli. I already have a mind set for sometime late September early October. When i feel really down about this LONG wait.... i think about the thousands of kids that can't even take PTC124 and they don't have anything so real ... so soon... then i said ... let just wait a little longer... we are in a very "lucky" group.
God bless.... Jose, Angie, Daniel and the Castro Gang
Joanne Wechlser said:Hi Jose, etc,
We too are waiting for the trial to start in Philadelphia. It's now August 23rd and still waiting. We did get an e-mail from Teesha the other day, but she hasn't responded as to when we will get our initial screening bloodkit in the mail. We're anxiously awaiting some kind of schedule or at least more information. Have you heard anything?
Joanne
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Permalink Reply by Angela Ryan on
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Our son is in the Phase 2B study in Philadelphia. He has been on the drug/placebo (??) for two weeks as of today. We've noticed no change. Did any one notice a physical change when their boys where in the Phase 2a study? If so, how soon was it before you noticed a difference?
Angela
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Hello Angela, Daniel took PTC124 about 2 years ago for 28 days and we really did not noticed any meaninful physical changes during thosse days. AS you might know there were reports from other boys showing some improvement but our Daniel didn't show it. Keep in mind that they weren't expecting any physical improvements during that study since the purpose of it was to check for safety and those results were great. None of the 38 kids showed any strong side effects. Most importantly ALL the kids produce dystrhophyne in-vitro. We are starting next week to get his EKG test doen in preparation to go to Phili soon. We just can't wait.
God bless you ALL
The Castro Gang
Angela Ryan said:Our son is in the Phase 2B study in Philadelphia. He has been on the drug/placebo (??) for two weeks as of today. We've noticed no change. Did any one notice a physical change when their boys where in the Phase 2a study? If so, how soon was it before you noticed a difference?
Angela
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Hi Jose and gang,
Just checking in again. Do you have dates? It's Sept 10th now and we're still waiting. I've been staying in touch with them, but still not getting anything definitive. So yes, my patience is getting tired, as now we are into school and a million other activities. You're right we are lucky. We had my son's echo and ekg done in July in anticipation of an August date. Are you in the Philly area or will you be traveling? Have you started with the other screening with the blood work?
We're hanging in there, but a little anxious. My son is 12 already.
Joanne
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Hi Angela,
We had one day while on PTC 2 years ago where my son was able to walk much much further than he had the previous year. It was an annual walk the school does, so we had something to compare it to. He walked almost 3 miles. The year prior he only walked about 1. Purely anecdotal. Unfortunately my son also had some additional headaches while taking the drug. Best of luck. I know this trial is hard on all of us. But on the flip side, provides so much hope.
Joanne
dacas01 said:Hello Angela, Daniel took PTC124 about 2 years ago for 28 days and we really did not noticed any meaninful physical changes during thosse days. AS you might know there were reports from other boys showing some improvement but our Daniel didn't show it. Keep in mind that they weren't expecting any physical improvements during that study since the purpose of it was to check for safety and those results were great. None of the 38 kids showed any strong side effects. Most importantly ALL the kids produce dystrhophyne in-vitro. We are starting next week to get his EKG test doen in preparation to go to Phili soon. We just can't wait.
God bless you ALL
The Castro Gang
Angela Ryan said:Our son is in the Phase 2B study in Philadelphia. He has been on the drug/placebo (??) for two weeks as of today. We've noticed no change. Did any one notice a physical change when their boys where in the Phase 2a study? If so, how soon was it before you noticed a difference?
Angela
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Thanks for the information. I didn't really expect to see a change yet. I was so surprised at the improvement he had with the Deflazacort when he was 4 that I was hoping we would see a similar response to this. I'll just have to wait and see.
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Just checking in again. Do you have dates? It's Sept 10th now and we're still waiting. I've been staying in touch with them, but still not getting anything definitive. So yes, my patience is getting tired, as now we are into school and a million other activities. You're right we are lucky. We had my son's echo and ekg done in July in anticipation of an August date. Are you in the Philly area or will you be traveling? Have you started with the other screening with the blood work?
We're hanging in there, but a little anxious. My son is 12 already.
Joanne, we are very much in the same situation. We are still waiting. We got his echo and EKG a couple of days ago. No dates just waiting. I want to keep possitive and think that we have awaited over 2 years jsut a few more weeks doesn't look that long. My son is 10 and he stoped walking about 4 or 5 months ago. Trust me we want this yesterday, but unfortunately is out of our control. I don't want to get upset with Dr. Finkell and his team since i feel they are doing things as fast as the protocol permits. All i can tell you, coming from someone in the exact same position you are, lets try to be a little bit more patient, it will happen before we know it. We live in South Florida and will have to do a lot of traveling for the procedures, but that is a very small price. No blood work done as of today yet. As soon as i know more i will post it here.
Best Regards
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Please ignore my previous post i made a mistake..... Sorry
Joanne, we are very much in the same situation. We are still waiting. We got his echo and EKG a couple of days ago. No dates just waiting. I want to keep possitive and think that we have awaited over 2 years jsut a few more weeks doesn't look that long. My son is 10 and he stoped walking about 4 or 5 months ago. Trust me we want this yesterday, but unfortunately is out of our control. I don't want to get upset with Dr. Finkell and his team since i feel they are doing things as fast as the protocol permits. All i can tell you, coming from someone in the exact same position you are, lets try to be a little bit more patient, it will happen before we know it. We live in South Florida and will have to do a lot of traveling for the procedures, but that is a very small price. No blood work done as of today yet. As soon as i know more i will post it here.
Best Regards
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