Kalydeco or VX-770 off lable use?

[saveferris2009]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>Rummage</b></i> http://chestjournal.chestpubs.org/content/early/2012/02/29/chest.11-2672.abstract</end quote>

No no no no no.
This is the Phase II trial - I'm looking for the Phase III trial data.
I don't think it's published... Ithink it may have just been a poster presented at NACF with data on file.
The data showed a significant impact on patients with DDF508.

 

[saveferris2009]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>Rummage</b></i> http://chestjournal.chestpubs.org/content/early/2012/02/29/chest.11-2672.abstract</end quote>

No no no no no.
This is the Phase II trial - I'm looking for the Phase III trial data.
I don't think it's published... Ithink it may have just been a poster presented at NACF with data on file.
The data showed a significant impact on patients with DDF508.

 

[Rummage]

I gave the link to the poster from the NACFC this year. If they are continuing this study in a phase III, I will look forward to reading about it.

 

[Rummage]

I gave the link to the poster from the NACFC this year. If they are continuing this study in a phase III, I will look forward to reading about it.

 

[Aboveallislove]

My understanding is that 770 did not do Phase 3 for dd508. Here is the summary of the research on 770 from cff.org which seems to confirm that:
http://www.cff.org/research/DrugDevelopmentPipeline/

 

[Aboveallislove]

My understanding is that 770 did not do Phase 3 for dd508. Here is the summary of the research on 770 from cff.org which seems to confirm that:
http://www.cff.org/research/DrugDevelopmentPipeline/

 

[musclemania70]

this link shows the completion date for phase 3 770 for dd508 to be July 2012
http://clinicaltrials.gov/ct2/show/NCT00953706?term=vertex+cystic+fibrosis&rank=11


this is from a press release dated June 2011
DISCOVER

The Phase 2 DISCOVER study (n=140) was primarily designed to provide additional safety data for VX-770 as part of the registration program. DISCOVER enrolled people 12 years and older with two copies of the F508del mutation, which prevents the CFTR protein from moving to its proper location at the cell surface. The majority of people with CF have at least one copy of the F508del mutation. All patients, including those in the placebo group, who completed study treatment in DISCOVER and met other criteria, were eligible to receive VX-770 as part of an extension study called PERSIST. Study investigators were notified in May 2011 to discontinue treating patients from DISCOVER who had enrolled in PERSIST because the improvements in lung function observed in DISCOVER were not considered clinically meaningful. Final data from this study were presented yesterday at the 34th European Cystic Fibrosis Conference in Hamburg, Germany.

 

[musclemania70]

this link shows the completion date for phase 3 770 for dd508 to be July 2012
http://clinicaltrials.gov/ct2/show/NCT00953706?term=vertex+cystic+fibrosis&rank=11


this is from a press release dated June 2011
DISCOVER

The Phase 2 DISCOVER study (n=140) was primarily designed to provide additional safety data for VX-770 as part of the registration program. DISCOVER enrolled people 12 years and older with two copies of the F508del mutation, which prevents the CFTR protein from moving to its proper location at the cell surface. The majority of people with CF have at least one copy of the F508del mutation. All patients, including those in the placebo group, who completed study treatment in DISCOVER and met other criteria, were eligible to receive VX-770 as part of an extension study called PERSIST. Study investigators were notified in May 2011 to discontinue treating patients from DISCOVER who had enrolled in PERSIST because the improvements in lung function observed in DISCOVER were not considered clinically meaningful. Final data from this study were presented yesterday at the 34th European Cystic Fibrosis Conference in Hamburg, Germany.

 

[Rummage]

I have a feeling there is some confusion because the G551D groups (Strive and Envision) also went into the PERSIST study extension and the results from Strive plus Persist were shown in poster 204 from the meeting. Again, if there is any more information out there, I am would love to read about it!

 

[Rummage]

I have a feeling there is some confusion because the G551D groups (Strive and Envision) also went into the PERSIST study extension and the results from Strive plus Persist were shown in poster 204 from the meeting. Again, if there is any more information out there, I am would love to read about it!

 

[musclemania70]

I read all the data from poster 204 and 206. No mention at all about a group of homozygous F508s with an increase in Fev1.
Poster 204 groups everyone together. He/she was right. There is a chart that shows change in fev1 but it does not break down the change in fev1 according to mutation.

 

[musclemania70]

I read all the data from poster 204 and 206. No mention at all about a group of homozygous F508s with an increase in Fev1.
Poster 204 groups everyone together. He/she was right. There is a chart that shows change in fev1 but it does not break down the change in fev1 according to mutation.

 

[Aboveallislove]

It is in the pdf Chest article, page 16 and 17. I do not see it in the Poster either. Have lots of thoughts on it and will send this weekend. Also will see if I can figure out if dd508 went to phase 3. . . I thought they didn't, but will investigate based on above. Thanks for pdf!!!!

 

[Aboveallislove]

It is in the pdf Chest article, page 16 and 17. I do not see it in the Poster either. Have lots of thoughts on it and will send this weekend. Also will see if I can figure out if dd508 went to phase 3. . . I thought they didn't, but will investigate based on above. Thanks for pdf!!!!