Impact of Discontinuing Chronic Therapies in People With Cystic Fibrosis on Highly Effective CFTR Modulator Therapy
(SIMPLIFY Trial)
Trial Summary
What is the purpose of this trial?
Despite the increasingly common use of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies in treating CF, it is still largely unknown whether or not other chronic therapies can be safely stopped. The SIMPLIFY study is being done to test whether or not it is safe to stop taking inhaled hypertonic saline or Pulmozyme® (dornase alfa) in those people that are also taking Trikafta™. Trikafta (elexacaftor/tezacaftor/ivacaftor) is a combination CFTR modulator therapy that was approved by the Food and Drug Administration for people with CF who have at least one F508del mutation. The three drugs that make up Trikafta work together to allow many more chloride ions to move into and out of the cells, improving the balance of salt and water in the lungs. These changes result in better clearance of mucus from the lungs and improvements in lung function. Inhaled hypertonic saline and Pulmozyme (dornase alfa) also improve clearance of mucus from the lungs to support lung function and have been available to people with CF for many years. Both therapies are considered to be relatively burdensome and it is not known whether either therapy can improve or maintain lung function above what is already gained through Trikafta use. The goal of the SIMPLIFY study is to get information about whether or not it is safe to stop either inhaled hypertonic saline or Pulmozyme (dornase alfa) by testing if there is a change in lung function in subjects with cystic fibrosis (CF) who are assigned to stop their chronic medication (either hypertonic saline or Pulmozyme) as compared to those who are assigned to keep taking their medication while continuing to take Trikafta.
Research Team
Nicole Mayer-Hamblett, PhD
Principal Investigator
University of Washington/Seattle Children's
Alex Gifford, MD, FCCP
Principal Investigator
Dartmouth-Hitchcock Medical Center
Eligibility Criteria
Inclusion Criteria
Treatment Details
Interventions
- Continuation of dornase alfa (dnase) (Other)
- Continuation of hypertonic saline (HS) (Other)
- Discontinuation of dornase alfa (dnase) (Other)
- Discontinuation of hypertonic saline (HS) (Other)
- Trikafta (CFTR Modulator Therapy)
Trikafta is already approved in Canada for the following indications:
- Cystic fibrosis in patients aged 2 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive based on in vitro data
Find a Clinic Near You
Who Is Running the Clinical Trial?
David Nichols, MD
Lead Sponsor
Nicole Hamblett
Lead Sponsor
University of Washington
Collaborator
Dr. Timothy H. Dellit
University of Washington
Chief Executive Officer since 2023
MD from University of Washington
Dr. Anneliese Schleyer
University of Washington
Chief Medical Officer since 2023
MD, MHA
Cystic Fibrosis Foundation
Collaborator
Michael P. Boyle
Cystic Fibrosis Foundation
Chief Executive Officer since 2019
MD from Johns Hopkins University
Albert Faro
Cystic Fibrosis Foundation
Chief Medical Officer since 2023
MD
Dartmouth-Hitchcock Medical Center
Collaborator
Jonathan T. Huntington
Dartmouth-Hitchcock Medical Center
Chief Medical Officer since 2024
MD, PhD, MPH
Joanne M. Conroy
Dartmouth-Hitchcock Medical Center
Chief Executive Officer since 2017
MD from Medical University of South Carolina