Impact of Discontinuing Hypertonic Saline in People With CF on Highly Effective CFTR Modulators- A SIMPLIFY Sub-Study
NCT ID: NCT06350461
Last Updated: 2024-10-01
Study Results
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View full resultsBasic Information
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COMPLETED
NA
370 participants
INTERVENTIONAL
2020-08-25
2022-07-11
Brief Summary
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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).
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Detailed Description
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Clinical outcomes (forced expiratory volume in 1 second \[FEV1\], antibiotic use, pulmonary exacerbations, and patient reported outcomes), safety (adverse events) and patient reported outcomes to evaluate respiratory symptoms and the participant's perception of how stopping HS would impact their daily life will be evaluated in all subjects. Additionally, a subset of participants at selected study sites will participate in Multiple Breath Washout (MBW) to evaluate changes in lung clearance index (LCI).
Conditions
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Study Design
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RANDOMIZED
PARALLEL
OTHER
NONE
Study Groups
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HS-Discontinue
Discontinuation of current hypertonic saline (HS) therapy
Discontinuation of hypertonic saline (HS)
Discontinuation of current hypertonic saline (HS) therapy during 6-week study period.
HS-Continue
Continuation of current hypertonic saline (HS) therapy
Continuation of hypertonic saline (HS)
Continuation of current hypertonic saline (HS) therapy during 6-week study period. Therapy is taken at least once daily according to each participant's pre-existing, clinically prescribed regimen (e.g., daily, twice daily). The concentration of HS is according to clinical prescription (e.g., 7% sodium chloride or 3.5% sodium chloride) and at least 3%.
Interventions
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Discontinuation of hypertonic saline (HS)
Discontinuation of current hypertonic saline (HS) therapy during 6-week study period.
Continuation of hypertonic saline (HS)
Continuation of current hypertonic saline (HS) therapy during 6-week study period. Therapy is taken at least once daily according to each participant's pre-existing, clinically prescribed regimen (e.g., daily, twice daily). The concentration of HS is according to clinical prescription (e.g., 7% sodium chloride or 3.5% sodium chloride) and at least 3%.
Eligibility Criteria
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Inclusion Criteria
* 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.
Exclusion Criteria
* 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.
12 Years
ALL
No
Sponsors
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Cystic Fibrosis Foundation
OTHER
Dartmouth-Hitchcock Medical Center
OTHER
University of Washington
OTHER
Nicole Hamblett
OTHER
Responsible Party
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Nicole Hamblett
Professor of Pediatrics, Division of Pulmonary and Sleep Medicine, University of Washington School of Medicine Adjunct Professor, Biostatistics, University of Washington School of Medicine Co-Executive Director, Cystic Fibrosis Therapeutics Development
Principal Investigators
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Nicole Mayer-Hamblett, PhD
Role: PRINCIPAL_INVESTIGATOR
University of Washington/Seattle Children's
Alex Gifford, MD, FCCP
Role: PRINCIPAL_INVESTIGATOR
Dartmouth-Hitchcock Medical Center
Locations
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University of Alabama at Birmingham
Birmingham, Alabama, United States
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 Iowa
Iowa City, Iowa, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
University of Kentucky
Lexington, Kentucky, United States
University of Louisville
Louisville, Kentucky, United States
Tulane University
Metairie, Louisiana, United States
Maine Medical Partners Pediatric Specialty Care
Portland, Maine, United States
Johns Hopkins University
Baltimore, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
University of Michigan, Michigan Medicine
Ann Arbor, Michigan, United States
Wayne State University Harper University Hospital
Detroit, Michigan, United States
Corewell Health Helen DeVos
Grand Rapids, Michigan, United States
Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, United States
The Minnesota Cystic Fibrosis Center
Minneapolis, Minnesota, United States
Children's Mercy Kansas City
Kansas City, Missouri, United States
Washington University School of Medicine
St Louis, Missouri, United States
Billings Clinic
Billings, Montana, United States
Dartmouth Hitchcock Medical Center
Lebanon, New Hampshire, United States
Monmouth Medical Center
Eatontown, New Jersey, United States
Morristown Medical Center
Morristown, New Jersey, United States
Rutgers - Robert Wood Johnson Medical School
New Brunswick, New Jersey, United States
Cohen Children's Medical Center of New York
New Hyde Park, New York, United States
Beth Israel Medical Center
New York, New York, United States
Lenox Hill Hospital Cystic Fibrosis Center
New York, New York, United States
Columbia University Cystic Fibrosis Program
New York, New York, United States
University of Rochester Medical Center Strong Memorial
Rochester, New York, United States
SUNY Upstate Medical University
Syracuse, New York, United States
New York Medical College at Westchester Medical Center
Valhalla, New York, United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States
Atrium Health Wake Forest Baptist
Winston-Salem, North Carolina, United States
Children's Hospital Medical Center of Akron
Akron, Ohio, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States
Cleveland Clinic Cystic Fibrosis Program
Cleveland, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Dayton Children's Hospital
Dayton, Ohio, United States
Oregon Health & Sciences University
Portland, Oregon, United States
Hershey Medical Center Pennsylvania State University
Hershey, Pennsylvania, United States
University of Pennsylvania
Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
Dell Children's Medical Center of Central Texas
Austin, Texas, United States
University of Texas Southwestern / Children's Health
Dallas, Texas, United States
University of Texas Southwestern
Dallas, Texas, United States
Cook Children's Medical Center
Fort Worth, Texas, United States
University of Texas Health Center at Tyler
Tyler, Texas, United States
Primary Children's Cystic Fibrosis Center
Salt Lake City, Utah, United States
University of Vermont Medical Center
Burlington, Vermont, United States
University of Virginia
Charlottesville, Virginia, United States
Virginia Commonwealth University
Richmond, Virginia, United States
Seattle Children's Hospital
Seattle, Washington, United States
University of Washington Medical Center
Seattle, Washington, United States
Providence Medical Group, Cystic Fibrosis Clinic
Spokane, Washington, United States
West Virginia University - Morgantown
Morgantown, West Virginia, United States
University of Wisconsin
Madison, Wisconsin, United States
Children's Wisconsin
Milwaukee, Wisconsin, United States
Froedtert & Medical College of Wisconsin
Milwaukee, Wisconsin, United States
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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SIMPLIFY-IP-19 HS
Identifier Type: -
Identifier Source: org_study_id
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