A new pilot study to examine the effect of Kalydeco on patients with evidence of residual CFTR function launched this month. The study sponsor is Vertex Pharma and the recruitment and study site is at National Jewish Health in Denver, Colorado.
Here is the official description: "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."
The layperson's key takeaway: if Ivacaftor/Kalydeco is shown to have a positive effect on lung function in patients with evidence of residual CFTR, this can potentially and significantly broaden the patient pool for whom Kalydeco could be of benefit. Our take: the study will show positive effects on lung function in patients with residual CFTR. We will have our eyes on the breakdown of effects by genotype.
The official link to the pilot study is here. They are recruiting candidates now, and if you qualify, and particularly if you are in the Denver area, contact Vertex at 617.444.6777 or firstname.lastname@example.org, or contact us, and we'll help you get in touch.
In addition to encouraging as many qualified candidates to apply as possible, we are particularly interested in getting some IVS8-5T genotypes in the study, as this variant is carried by nearly 10% of the U.S. population and has a broad spectrum of phenotypic effects.
We will follow up later with a post on the significance of the 5T variant, as well as report on our progress trying to get the 5T added to the study.