Testing existing cystic fibrosis drugs for different gene mutations

This trial is testing whether three existing cystic fibrosis medications—Symdeko, Orkambi, and Ivacaftor—might work for people with cystic fibrosis who have gene mutations different from the ones these drugs are currently approved to treat. First, the research team will study your specific mutation in the laboratory to see which medication might help your cells work better. Then, based on those lab results, you'll receive the medication that appears most likely to benefit your particular mutation.

Many people with cystic fibrosis have rare gene mutations that are not yet approved for any treatment. This trial explores whether medications that are already proven safe and effective for other cystic fibrosis mutations might also help people with these rarer mutations, potentially offering new treatment options where few currently exist.