Clinical Trial to Keep Our Eyes On

This clinical trial update recently came out and I've been meaning to share it with all of you!

In layman terms, this clinical trial is testing the effectiveness of Kalydeco™ paired with a second drug, VX-661, for people with one copy of DF508 and another mutation showing residual function, aged 12 or older.  They are currently recruiting individuals that fit this criteria across 70 CF care locations.  This phase of the trial is expected to run through spring 2016.

So what exactly does phase III mean when it comes to clinical trials?  According to the National Library of Medicine, phase III testing means the drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.

If successful, this will likely move to phase IV which is the final step before making the combo drug available to the masses!  If all goes well, we can hope they begin to test the effectiveness in a younger age group (2 - 12) following the release to those 12 and older.  Fingers crossed.  This. Is. Huge.

UNC Chapel Hill is one of the participating locations for this clinical trial, which is not surprising as they are a leader in CF care and research.  We have been contemplating making a shift in Sienna's care due to the very passive approach her current team takes and even have a referral out to another Pulmonologist in the Charlotte area.  With this news, I'm wondering if making the shift to a Pulmonologist at UNC Chapel Hill would be the best move.  This can potentially get her into a clinical trial and speed up the process of getting key medicines like the combo drug mentioned above (obviously there is no guarantee to get the combo drug during a trial, as a portion of participants are placed on a placebo).

We are at least a year out from exploring the option of participating in a trial...but it's exciting to know that these clinical trials are happening!  The progress made over the past two years is incredibly encouraging to see as parents of a young child with a life shortening illness that has no cure.  There is so much hope!  A big THANK YOU to all of you who continue to support this cause close to our heart by donating and raising awareness.

Enjoying Easter Sunday

iPad time at clinic

Easter egg hunt!