Vertex Pharmaceuticals initiates Phase IIa cystic fibrosis trial

Vertex Pharmaceuticals, a biotechnology company, has initiated a Phase IIa clinical trial for VX-809, an investigational cystic fibrosis transmembrane conductance regulator corrector that targets the defective CFTR protein that causes cystic fibrosis.

The trial is designed primarily to evaluate the safety and tolerability of multiple doses of VX-809 in patients with cystic fibrosis (CF). In addition to assessing safety, the trial will also evaluate the potential effect of VX-809 on measures of cystic fibrosis transmembrane conductance regulator (CFTR) function.

The Phase IIa trial for VX-809 announced is expected to enroll approximately 90 patients with CF ages 18 years or older who are homozygous for the F508del CFTR mutation. The trial will be conducted at approximately 22 clinical trial sites in North America and Europe. Patient screening is now underway at certain clinical trial sites.

The randomized, double-blind study will initially evaluate two dose levels of VX-809 compared to placebo given orally once daily for 28 days in a parallel design. Following a planned interim safety analysis, the trial may evaluate an additional two dose levels of VX-809 compared to placebo. The trial is expected to be complete in early 2010.

Freda Lewis-Hall, chief medical officer of Vertex, said: This Phase IIa trial is designed primarily to provide a safety, tolerability and pharmacokinetic evaluation for VX-809. Any additional signals observed in this trial, including changes in sweat chloride and nasal potential difference, or any measurable impact on FEV1, that indicate VX-809 has an effect on measures of CFTR function may be highly informative and encouraging for planning future clinical trials with VX-809.