Talissa A. Altes1, Mac Johnson2, John P. Mugler1, Deborah Froh3, Lucia Flors1, Grady Wilson Miller1, Jaime H. Mata1, Carlos L. Salinas1, Nick Tustison1, Po-Shun Lee2, Tao Song2, Karl Yen2, Eduard E. de Lange1, Martyn C. Botfield2
1Radiology, University of Virginia, Charlottesville, VA, United States; 2Vertex Pharmaceuticals Incorporated, Cambridge, MA, United States; 3Pediatric Pulmonology, University of Virginia, Charlottesville, VA, United States
This study to evaluate the ability of hyperpolarized helium-3 MR ventilation imaging to detect the effects of treatment with ivacaftor, a CFTR potentiator, demonstrated a rapid onset of improvement in lung ventilation with treatment and equally rapid return to near baseline ventilation following cessation of treatment. Eight subjects with CF were imaged on 5 different days, 2 weeks apart (Days 0, 14, 28, 42, and 56), and were treated with Ivacaftor from Days 14 to 42. Interim data analysis has been conducted on the first 4 subjects in the study, although all 8 subjects have completed the trial.