052-20 | Impact of Discontinuing Chronic Therapies in People With Cystic Fibrosis on Highly Effective CFTR Modulator Therapy

Overview

“A master protocol to test the impact of discontinuing chronic therapies in people with cystic fibrosis on highly effective CFTR modulator therapy (SIMPLIFY)”

Health outcomes in people with Cystic Fibrosis have steadily improved for decades through high quality, multidisciplinary clinical care models and expanding therapeutic options. These advances have most often been additive and require people with CF and their families to dedicate significant time and resources toward daily care needs. Since 2012, a small but increasing number of people with CF have benefited from highly effective CFTR modulator treatment (HEMT). As transformative modulator drug therapies emerge, many in the CF Community (patients, families and caregivers) are asking if any of the pre-existing therapies can be reduced or eliminated. Transformative improvement in CF clinical care through HEMT represents an important opportunity to test the impact of withdrawing chronic therapies, specifically hypertonic saline and dornase alfa, in a randomized, controlled setting. The SIMPLIFY master protocol will study people treated with HEMT who also use inhaled hypertonic saline and/or inhaled dornase alfa by clinical prescription. In this adolescent and adult study population, we will be testing the question of treatment discontinuation. As there are currently no data from controlled trials testing the effects of discontinuing these therapies in CF, the results of this protocol will be important when considering individual treatment decisions among the increasingly large percentage of people with CF on highly effective modulator therapies.