Things have finally cleared up on the path forward for those that have type IV (sans G551D) or V mutations in regards toIvacaftor. Four studies have started: three for phase III trial for type IV mutations and another for select type V mutations.
For those with a mutation in one of these 2 classes, it has been a frustrating couple of years as there has been very little news about how Vertex and CFFT would move forward with VX-770 and these defects. From the limited in-vitro studies, it has always been clear that Ivacaftor could have the potential to make a clinical difference if it could only be tested. With the very high price, off-labeling is unlikely for those impacted with waiting for clinical trials so waiting for any trial news is all those impacted by these defects.
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How things have changed. Not too long ago, it was not clear how Vertex was planning on moving forward. There were discussions on doing n-of-1 trials but that seems to be a distant memory (how likely was it to get the FDA to approve a non-standard approach?). Now several trials are started and it looks like now all we have to do is wait and keep our fingers crossed that they all meet their end point measurements. The interesting note here is that the trail for residual CFTR function is only a phase II trial meaning that we are many years away, best case, to have the drug approved for use with their defect. It’s hard to understand why a phase II trial is needed considering a phase II was already completed for those with normal CFTR function and those with no function. Are they are expecting different results or is this the FDA at their best once again? The other possibility is they want to see if there is a significant change in lung function. One could speculate that the baseline of FEV1 is already high and because these defects are not gating, the amount that Ivacaftor can help will not be nearly as significant. It may require many more subtle measurements to know if Ivacaftor should be approved for this class of defects. This is where it would be really nice if the CFF organization could be more open about the process.
Below is a list of the clinical trials with their primary completion dates:
Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis and Residual CFTR Function completes September 2013
Study of Ivacaftor in Subjects With Cystic Fibrosis Who Have the R117H-CFTR Mutation (KONDUCT) complets July 2013
Rollover for the above trials: Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation (KONTINUE) completes July 2016