CHaractErizing CFTR Modulated Changes in Sweat Chloride and Their Association With Clinical Outcomes

NCT ID: NCT03350828

Last Updated: 2025-10-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

5000 participants

Study Classification

OBSERVATIONAL

Study Start Date

2018-01-15

Study Completion Date

2026-12-31

Brief Summary

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This is a multicenter, cross-sectional, cohort study which will collect contemporary sweat chloride (SC) values from approximately 5000 Cystic Fibrosis (CF) patients prescribed and currently receiving commercially approved Cystic Fibrosis transmembrane conductance regulator (CFTR) modulator therapies.

Detailed Description

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Eligible subjects who have been prescribed and chronically taking a commercially approved CFTR modulator for at least 3 months will be enrolled for a single visit to collect sweat to be analyzed for SC at their local site laboratory. Limited clinical data obtained at this visit will be augmented by retrospective and prospective data obtained from the Cystic Fibrosis Foundation Patient Registry (CFFPR). Study subjects who have been prescribed and switch to an alternative commercially approved CFTR modulator will be approached to re-enroll in the study after being on the alternative modulator for at least 3 months so that a new SC value can be obtained.

Conditions

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Cystic Fibrosis

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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Inclusion Criteria

1. Written informed consent (and assent when applicable) obtained from subject or subject's legal representative
2. Enrolled in the CFFPR
3. Male or female ≥ 4 months of age on day of study visit
4. Diagnosis of CF.
5. Current treatment with a prescribed commercially approved CFTR modulator for at least 90 days prior to enrollment
6. Able to perform the testing and procedures required for this study, as judged by the investigator


1. Male or female ≥ 6 years of age on day of study visit.
2. Current treatment with elexacaftor/tezacaftor/ivacaftor for at least 90 days prior to enrollment.
3. Last dose of elexacaftor/tezacaftor/ivacaftor taken at least 24hours and last dose of ivacaftor taken at least 12 hours prior to trough blood draw on day of visit.

Exclusion Criteria

1. Presence of a condition or abnormality that, in the opinion of the Investigator, would compromise the safety of the patient or the quality of the data
2. Currently enrolled in an investigational trial (including open-label follow-on studies and Early Access Programs (EAP) of an agent expected to have an impact on sweat chloride (refer to current list provided on study website)
Minimum Eligible Age

4 Months

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Cystic Fibrosis Foundation

OTHER

Sponsor Role collaborator

Nicole Hamblett

OTHER

Sponsor Role lead

Responsible Party

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Nicole Hamblett

Professor in the Department of Pediatrics in the Division of Pulmonary Medicine

Responsibility Role SPONSOR_INVESTIGATOR

Principal Investigators

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Edith Zemanick, MD

Role: PRINCIPAL_INVESTIGATOR

University of Colorado, Denver

Michael Konstan, MD

Role: PRINCIPAL_INVESTIGATOR

Case Western Reserve University

Nicole Mayer-Hamblett, PhD

Role: PRINCIPAL_INVESTIGATOR

Seattle Children's Hospital

Locations

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The Children's Hospital Alabama, University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status

University of Arkansas for Medical Sciences

Little Rock, Arkansas, United States

Site Status

University of California San Diego

La Jolla, California, United States

Site Status

Childrens Hospital Los Angeles

Los Angeles, California, United States

Site Status

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status

National Jewish Health

Denver, Colorado, United States

Site Status

Yale University School of Medicine

New Haven, Connecticut, United States

Site Status

University of Florida

Gainesville, Florida, United States

Site Status

The Nemours Children's Clinic - Orlando

Orlando, Florida, United States

Site Status

Nemours Children's Clinic - Pensacola

Pensacola, Florida, United States

Site Status

Children's Healthcare of Atlanta and Emory University

Atlanta, Georgia, United States

Site Status

Emory University

Atlanta, Georgia, United States

Site Status

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Site Status

University of Chicago

Chicago, Illinois, United States

Site Status

Riley Hospital for Children

Indianapolis, Indiana, United States

Site Status

University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

University of Kentucky

Lexington, Kentucky, United States

Site Status

John Hopkins Hospital

Baltimore, Maryland, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

University of Michigan, Michigan Medicine

Ann Arbor, Michigan, United States

Site Status

Children's Hospital of Michigan

Detroit, Michigan, United States

Site Status

Helen DeVos Children's Hospital

Grand Rapids, Michigan, United States

Site Status

The Minnesota Cystic Fibrosis Center

Minneapolis, Minnesota, United States

Site Status

Children's Mercy Kansas City

Kansas City, Missouri, United States

Site Status

SSM Health Cardinal Glennon Children's Hospital

St Louis, Missouri, United States

Site Status

St. Louis Children's Hospital

St Louis, Missouri, United States

Site Status

Dartmouth Hitchcock Medical Center

Lebanon, New Hampshire, United States

Site Status

The Cystic Fibrosis Center of Western New York

Buffalo, New York, United States

Site Status

New York Medical College at Westchester Medical Center

Valhalla, New York, United States

Site Status

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Site Status

Children's Hospital Medical Center of Akron

Akron, Ohio, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center

Cleveland, Ohio, United States

Site Status

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

Toledo Children's Hospital

Toledo, Ohio, United States

Site Status

Oregon Health Sciences University

Portland, Oregon, United States

Site Status

Hershey Medical Center Pennsylvania State University

Hershey, Pennsylvania, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

Site Status

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

University of Tennessee CF Care and Research Center

Memphis, Tennessee, United States

Site Status

Vanderbilt Children's Hospital

Nashville, Tennessee, United States

Site Status

University of Texas Southwestern / Children's Health

Dallas, Texas, United States

Site Status

University of Texas Southwestern Medical Center

Dallas, Texas, United States

Site Status

Baylor College of Medicine

Houston, Texas, United States

Site Status

Primary Children's Cystic Fibrosis Center

Salt Lake City, Utah, United States

Site Status

Seattle Children's Hospital

Seattle, Washington, United States

Site Status

University of Washington Medical Center

Seattle, Washington, United States

Site Status

West Virginia University - Morgantown

Morgantown, West Virginia, United States

Site Status

University of Wisconsin

Madison, Wisconsin, United States

Site Status

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

Countries

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United States

References

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VanDevanter DR, Zemanick ET, Konstan MW, Ren CL, Odem-Davis K, Emerman I, Young J, Mayer-Hamblett N; CHEC-SC Study Group. Willingness of people with cystic fibrosis receiving elexacaftor/tezacaftor/ivacaftor (ETI) to participate in randomized modulator and inhaled antimicrobial clinical trials. J Cyst Fibros. 2023 Jul;22(4):652-655. doi: 10.1016/j.jcf.2023.04.007. Epub 2023 Apr 24.

Reference Type DERIVED
PMID: 37100705 (View on PubMed)

Other Identifiers

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CHEC-OB-17

Identifier Type: -

Identifier Source: org_study_id

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