Is anyone currently enrolled in the phase II or phase IIa PTC 124 trial? We are currently finishing the pre-approval process with getting a biopsy next week and then onto the medicine Feb. 1. Looking to see if anyone else has been in the ongoing or the new to start one.


Matt

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Look up Angela Knight. Her son is in the trial.
Thanks I tried to send her a message but it won't let me unless I am her friend. Not sure why she would friend someone she has never met or spoke to before. Hopefully she will see this and understand the request so I can speak to her.

Liisa Underwood said:
Look up Angela Knight. Her son is in the trial.
Our son Elliott is currently in the Phase 2B Extension study...

What trial are you enrolling in? The only one I am aware of that is currently enrolling is a Phase 2 study for boys who are no longer ambulatory. Is this the study you are enrolling in?

~P
I was under the impression that this trial is no longer enrolling patients. In fact it was full long time ago.
Actually - they just recently opened a new Phase 2 trial for boys who are no longer ambulatory...
www.clinicaltrials.gov - seach for ID NCT01009294

Ofelia Marin said:
I was under the impression that this trial is no longer enrolling patients. In fact it was full long time ago.
Matt, is this for the ambulatory trial which is currently in the final phase or the new trial for the non ambulatory trial? My son, Kyle, is in the ambulatory trial and are in final phase waiting for the FDA approval of ataluren (PTC124).

Donna
Yes our son is almost 13 and has been in a chair for almost 4 years now.

Paul Johnson said:
Our son Elliott is currently in the Phase 2B Extension study...

What trial are you enrolling in? The only one I am aware of that is currently enrolling is a Phase 2 study for boys who are no longer ambulatory. Is this the study you are enrolling in?

~P
Sorry I had that checked wrong our son is 12 almost 13. Non ambulatory study.

Ofelia Marin said:
Right. I do know about that. Matt's son is 0-3 (?) years old from what I see in his page... Also no trial enrolls boys younger than 5.

Paul Johnson said:
Actually - they just recently opened a new Phase 2 trial for boys who are no longer ambulatory...
www.clinicaltrials.gov - seach for ID NCT01009294

Ofelia Marin said:
I was under the impression that this trial is no longer enrolling patients. In fact it was full long time ago.
No Donna this is for the new non-ambulatory trial that is currently enrolling. We go next wednesday for his muscle biopsy, then start the medicine on the 1rst of February. Do you have an e-mail where I can contact you?


Donna said:
Matt, is this for the ambulatory trial which is currently in the final phase or the new trial for the non ambulatory trial? My son, Kyle, is in the ambulatory trial and are in final phase waiting for the FDA approval of ataluren (PTC124).

Donna

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