A Randomized, Double-Blind, Placebo-Controlled, Parallel Study to Evaluate Safety, Pharmacokinetics and Pharmacodynamics of FDL169 in Cystic Fibrosis Subjects Homozygous for the F508del-CFTR mutation

Project type/s Clinical trial
Project status Currently underway

Cystic fibrosis (CF) is a genetic disease characterized for a defective protein, called CFTR, which serves as a channel for the passage of charged ions, such as chloride, and it is involved in production of sweat, digestive fluids, and mucous from lungs and other organs. Mutations reducing either the amount of the protein at the cell surface, its ability to work as a chloride channel (channel gating mutations), or both, dysregulate salt and water absorption, and secretion of mucus in these tissues resulting in severe problems in the lung and other organs, ultimately reducing life expectancy.

Some preclinical studies have shown that the recently developed drug known as FDL169 has positive effects in restoring CFTR function in CF patients with two copies (homozygous) of the F508del mutation, but more research is required to fully evaluate FDL169’s safety and effectiveness in these patients. This project seeks to evaluate safety, pharmacokinetics and pharmacodynamics of FDL169 in Cystic Fibrosis individuals who are homozygous for the F508del-CFTR mutation.

Team Members

  • Dr Lucy Burr - Team Role: Principal Investigator
  • Megan Martin - Team Role: Project Coordinator