Impact of Discontinuing Hypertonic Saline in People With CF on Highly Effective CFTR Modulators- A SIMPLIFY Sub-Study

Completed · Not applicable

Conditions studied: Cystic Fibrosis

In brief

Despite the increasingly common use of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies in treating cystic fibrosis (CF), it is still largely unknown whether or not other chronic therapies can be safely stopped. This SIMPLIFY sub-study is being done to test whether or not it is safe to stop taking inhaled hypertonic saline in those people that are also taking elexacaftor/tezacaftor/ivacaftor (ETI). ETI 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 ETI 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 (HS) also improves clearance of mucus from the lungs to support lung function and has been available to people with CF for many years. HS is considered to be relatively burdensome and it is not known whether HS can improve or maintain lung function above what is already gained through ETI use. The goal of this SIMPLIFY sub-study is to get information about whether or not it is safe to stop hypertonic saline by testing if there is a change in lung function in participants with cystic fibrosis (CF) who are assigned to stop taking HS as compared to those who are assigned to keep taking HS while continuing to take ETI. This is a sub study of master protocol SIMPLIFY-IP-19, NCT04378153. The sub study investigating the impact of discontinuing and continuing dornase alfa is registered under NCTXXXXXXX (will add once available).

Key facts

Study ID

NCT06350461

Run by

Nicole Hamblett

People needed

370

Starts

2020-08-25

Expected to finish

2022-07-11

Last updated by the study team

2024-10-01

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%) 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 — 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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