WHAT is this study you are doing?

1st the Gobbeley-Gook.......English below

NOTE: Below is public information available at clinicaltrials.gov and anything outside of what is directly copied from the website is my interpretation and only my interpretation which may or may not be true and accurate. 

Official Title: A Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis, Residual CFTR Function, and FEV1 ≥40% Predicted

Brief Summary: This study is a multiple within subject crossover study to evaluate the effect of ivacaftor on lung function in subjects aged 12 years and older with cystic fibrosis (CF) who have phenotypic or molecular evidence of residual CF transmembrane conductance regulator (CFTR) function.

Official Title:

A Phase 3, Two-Part, Randomized, Double-Blind, Placebo-Controlled, Crossover Study With an Open-Label Period to Evaluate the Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Who Have a Non-G551D CFTR Gating Mutation

Kalydeco but also known as Ivacaftor, VX770, or affectionately "blue lightning" or just K is the focus of the study. K has been shown effective and is approved for use in cystic fibrosis patients with one specific gene mutation G551D which is only 4% of CF patients. This study looks at those mutations they think K may work on but that are too rare to do a full study on.

http://www.clinicaltrials.gov/ct2/show/NCT01685801?term=kalydeco&rank=17

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So for this study instead of trying to prove that K works for a certain subset of mutations they are instead trying to prove that they have a scientific way of showing who does and does not benefit from K. 

For example my mutation is estimated to have less than 200 people worldwide so they could never enroll enough people to do a study. In order to ‘prove’ something you have to have statistical significance which means a large sample of people to test. How then do you show that it works (or doesn't) and get it approved for use?

Thus they will give patients a trial of K and then see if it works w/in a certain period of time and what variables show an improvement compared to when they were only getting placebo. The way the study is set up would allow them to see by what point those who do react positively started having the improvements and what exactly showed the improvements since I am on K for a period of 2 weeks, 4 weeks, and 8 weeks during the study. 

So the set up of the study is:

    Note: You are randomly assigned to one of the study sequences and either way you do both drug and placebo; neither we nor the doctors know which sequence we are assigned to.

Phase 1 (2 Stages - each stage is 2 Weeks; one stage placebo, one stage drug)

• Kalydeco 2 weeks --> Placebo 2 weeks 

• Placebo  2 weeks --> Kalydeco 2 weeks

*4-8 WEEK BREAK to ‘wash out’ Drug; this serves to get us back to our normal baseline* 

Phase 2 (2 Stages - each stage is 4 weeks)

• Kalydeco 4 weeks --> Placebo  4 weeks 

• Placebo 4 weeks --> Kalydeco 4 weeks

*4-8 WEEK BREAK to ‘wash out’ Drug; this serves to get us back to our normal baseline* 

· Phase 3 (1 Stage – 8 weeks – everyone is ON)

There are 4 potential sequences: Kaly=ON; Placebo=OFF

·        2 weeks(ON then OFF) - 4 weeks(ON then OFF) --> 8 WEEKS ON

·        2 weeks(OFF then ON) - 4 weeks(OFF then ON) --> 8 WEEKS ON

·        2 weeks(ON then OFF) - 4 weeks(OFF then ON) --> 8 WEEKS ON

·        2 weeks(OFF then ON) - 4 weeks(ON then OFF) --> 8 WEEKS ON