A new study into a potential drug for cystic fibrosis has begun. The Phase Ib/IIa trial will investigate the drug POL6014 in up to forty patients who have cystic fibrosis. For more detailed information about this, you can view the source press release at Santhera’s website here.
About Cystic Fibrosis
Cystic fibrosis (CF) is an inherited genetic disorder that causes sticky mucus to build up in the lungs and digestive system. It’s caused by a genetic mutation that affects how salt and water are transported in and out of cells, which can cause mucus to build up. As a result, people with CF may experience problems with their lungs, a reduced ability to digest food, and frequent infections, amongst other symptoms. You can read more about CF here.
An investigational drug for CF, POL6014, is thought to work by inhibiting human neutrophil elastase. Previously, this drug has been tested in animal models, a Phase I trial of healthy volunteers, and a Phase Ia trial of patients with CF. These studies support the tolerability and strong effect of the drug, and based on these results, Santhera is now going ahead with a Phase Ib/IIa trial.
The Phase Ib/IIa Trial
The Phase Ib/IIa trial will be a multiple ascending dose trial of POL6014 in around forty patients with CF. People who take part will be treated with either POL6014 in one of three possible doses or a placebo once or twice a day for fifteen days. The trial, which is taking place in Germany and Poland, is designed to investigate how safe and tolerable the drug is, as well as its effects in the body. According to their announcement, Santhera expects the study to finish around the second half of 2019.