Clinical Trial for kids ages 6 and up with DD508

mbrandazzo

New member
Hi!

I was wondering if anyone has heard what is going on with the clinical trial for the combo of the Kalydeco/VX-809 for kids ages 6 and up. Our hospital was chosen as one of the sites and my son's doctor had mentioned it a few months ago... I followed up with her about it today and she said it had been put on hold. Does anyone know why? Thank you!
 

roeann

New member
Maybe its related to a clip from an article that I just read - based on discussions with researchers/doctors at the CF Society Conference in Sweden, analyst Geoff Porges has concluded that two pivotal phase III studies (TRAFFIC and TRANSPORT) for Kalydeco + VX809 is (more) likely to fail. The original piece is located at thestreet.com
 

rosesixtyfive

New member
Hi, roeann,
Where do you see an article about vertex not working. I went to thestreet.com and all I saw was an article from June 2nd that said they were going to soon announce results of the Phase III trial and that it could go either way.
Thanks
 

roeann

New member
The piece from the street wasn't real informative it was just waving a red flag. However, but a piece at fiercebiotech contains more details EG per Porges, Kalydeco shifts the temperature stability downward which counteracts the positive effect of Vx809 and the longer the exposure to Kalydeco (in a cell culture) the more the Vx809 CFTR correction 'erodes'.

The company originally said they would publish results in June but Porges says the date may be moved to July. Also of concern, the article suggests that these problems may impact delay VX-661.

Read more: http://www.fiercebiotech.com/story/...-crucial-combo-study/2014-06-16#ixzz351DAWSNo

Of course what happens in a lab doesn't necessarily happen in the 'real world'.. so I still have my fingers crossed.
 

Mallika

New member
I still have hopes. A comment for the article
: http://www.fiercebiotech.com/story/b...#ixzz351DAWSNo
reads that even Kalydeco had similar negative reviews when it came out. But look at the wonders it is doing. So hoping that it is the same with the Lumacaftor and Kalydeco also. Now waiting for July. Are there any other Pharmaceuticals researching on drugs for DDF
508 apart from N30 and Vertex?





 

jokamble

New member
Hi Mallika...i go to this forum mostly everyday and many years but somehow didn't write anything so far. After reading that you are from india I thought to write as we are also from India where CF is hardly known. My daughter is 17 years old with double delta 508.We are from Maharashtra ...from where do you come? And how is your daughter doing?
It's true that each and every person related to CF is waiting for the Vertex trial data and I too hope that it comes positive.
jyoti
 

Mallika

New member
Hi Jyoti,
Can not tell you how excited I am to talk to an other woman on this problem. The others parents I know in India are Kapil and Rahul fathers of CFers who are also on this forum. Yes I am from Bangalore. My Son is better now. Even I regularly go through this forum. I also visit vertex, N30 and Science Daily CF pages like a daily routine tying to understand this CF puzzle put before me. I am sending you my details in a message so that we can get in touch. BTW, India with its massive population is the biggest country with CF patients. Most of the cases here go misdiagnosed!
Mallika.
 
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