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

Completed

Conditions studied: Cystic Fibrosis

In brief

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.

Key facts

Study ID: NCT04378153
Run by: Nicole Hamblett
People needed: 987
Starts: 2020-08-25
Expected to finish: 2022-07-11
Last updated by the study team: 2024-12-04

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Diagnosis of CF.
Age ≥ 12 years at the Screening Visit.
Forced expiratory volume in 1 second (FEV1) ≥ 70 % predicted at the Screening Visit if < 18 years old, and ≥ 60 % predicted at Screening Visit if ≥ 18 years old.
Clinically stable with no significant changes in health status within the 7 days prior to and including the Screening Visit.
Current treatment with elexacaftor/tezacaftor/ivacaftor (ETI) for at least the 90 days prior to and including the Screening Visit and willing to continue daily use for the duration of the study.
Currently taking hypertonic saline (at least 3%) and/or dornase alfa for at least the 90 days prior to and including the Screening Visit and willing to continue daily use for the 2-week screening period.

You may not qualify if…

Active smoking or vaping.
Use of an investigational drug within 28 days prior to and including the Screening Visit.
Changes to chronic therapy (e.g., ibuprofen, azithromycin, inhaled tobramycin, aztreonam lysine) within 28 days prior to and including the Screening Visit. This includes new airway clearance routines.
Acute use of antibiotics (oral, inhaled or IV) or acute use of systemic corticosteroids for respiratory tract symptoms within 7 days prior to and including the Screening Visit.
Chronic use of systemic corticosteroids at a dose equivalent to ≥ 10mg per day of prednisone within 28 days prior to and including the Screening Visit.
Antibiotic treatment for nontuberculous mycobacteria (NTM) within 28 days prior to and including the Screening Visit.

Where it is running

Providence Alaska Medical Center — Anchorage, Alaska, United States
Tucson Cystic Fibrosis Center — Tucson, Arizona, United States
Arkansas Children's Hospital — Little Rock, Arkansas, United States
Miller Children's and Women's Hospital Long Beach — Long Beach, California, United States
CHOC Children's Hospital — Orange, California, United States
Stanford University Medical Center — Palo Alto, California, United States
Rady Children's Hospital and Health Center at the University of California San Diego — San Diego, California, United States
University of California, San Francisco - Adult Center — San Francisco, California, United States
University of California, San Francisco - Peds Center — San Francisco, California, United States
Children's Hospital Colorado — Aurora, Colorado, United States
National Jewish Health — Denver, Colorado, United States
Yale University School of Medicine — New Haven, Connecticut, United States
University of Florida — Gainesville, Florida, United States
Nemours Children's Clinic — Jacksonville, Florida, United States
Central Florida Pulmonary Group — Orlando, Florida, United States
The Nemours Children's Clinic - Orlando — Orlando, Florida, United States
Nemours Children's Clinic - Pensacola — Pensacola, Florida, United States
All Children's Hospital — St. Petersburg, Florida, United States
Tampa General Hospital — Tampa, Florida, United States
Emory University — Atlanta, Georgia, United States
Saint Luke's Cystic Fibrosis Center of Idaho — Boise, Idaho, United States
Northwestern University — Chicago, Illinois, United States
OSF Saint Francis Medical Center — Peoria, Illinois, United States
Riley Hospital for Children — Indianapolis, Indiana, United States
University of Alabama at Birmingham — Birmingham, Alabama, United States

Full record on ClinicalTrials.gov

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