CFF provided information on the following clinical trials:

09/04/2015Phase 1b safety study of QR-010 in adults with CF with two copies of the F508del-CFTR gene mutation

This is a phase 1b, multi center, placebo controlled trial that will look at the safety and tolerability of the inhaled drug QR-010. This trial will also look at how the body processes the drug.  Researchers will test the drug in people with cystic fibrosis who have two copies of the F508del-CFTR mutation. This new type of drug is designed to repair the RNA that creates the CFTR protein.

In this trial researchers will make sure that this drug is safe and look at how it works in the body when taken in different amounts. This trial will require blood draws, lung function tests, and sweat tests. It will last 8 weeks and is for people who are between the ages 18-60.

10/30/2015Phase 2 trial of CTX-4430 in people with CF

This is a phase 2 placebo controlled trial. It will look at the safety and effectiveness of the anti-inflammatory drug CTX-4430. Researchers aim to show the effectiveness and safety of CTX-4430 by showing improved lung function in those receiving the drug and fewer negative outcomes.

This trial is for people with CF ages 18 and older who have had a pulmonary exacerbation in the last year. It will require blood tests, lung function tests and monthly visits for 11 months.