CHaractErizing CFTR Modulated Changes in Sweat Chloride and Their Association With Clinical Outcomes (CHEC-SC)

The CHEC-SC Cohort Study: CHaractErizing CFTR Modulated Changes in Sweat Chloride and Their Association With Clinical Outcomes

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Cystic Fibrosis

Detailed Description

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.

• Study Type: Observational
• Enrollment (Estimated): 5000

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • The Children's Hospital Alabama, University of Alabama at Birmingham
  • Arkansas
    • Little Rock, Arkansas, United States, 72205
      • University of Arkansas for Medical Sciences
  • California
    • La Jolla, California, United States, 92093
      • University of California San Diego
    • Los Angeles, California, United States, 90027
      • Childrens Hospital Los Angeles
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Children's Hospital Colorado
    • Denver, Colorado, United States, 80206
      • National Jewish Health
  • Connecticut
    • New Haven, Connecticut, United States, 06520
      • Yale University School of Medicine
  • Florida
    • Gainesville, Florida, United States, 32610
      • University of Florida
    • Orlando, Florida, United States, 32827
      • The Nemours Children's Clinic - Orlando
    • Pensacola, Florida, United States, 32514
      • Nemours Children's Clinic - Pensacola
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Children's Healthcare of Atlanta and Emory University
    • Atlanta, Georgia, United States, 30327
      • Emory University
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Ann & Robert H. Lurie Children's Hospital of Chicago
    • Chicago, Illinois, United States, 60637
      • University of Chicago
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Riley Hospital for Children
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • University of Kansas Medical Center
  • Kentucky
    • Lexington, Kentucky, United States, 40506
      • University of Kentucky
  • Maryland
    • Baltimore, Maryland, United States, 21287
      • John Hopkins Hospital
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan, Michigan Medicine
    • Detroit, Michigan, United States, 48201
      • Children's Hospital of Michigan
    • Grand Rapids, Michigan, United States, 49503
      • Helen DeVos Children's Hospital
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • The Minnesota Cystic Fibrosis Center
  • Missouri
    • Kansas City, Missouri, United States, 64108
      • Children's Mercy Kansas City
    • St Louis, Missouri, United States, 63110
      • St. Louis Children's Hospital
    • St Louis, Missouri, United States, 63104
      • SSM Health Cardinal Glennon Children's Hospital
  • New Hampshire
    • Lebanon, New Hampshire, United States, 03756
      • Dartmouth Hitchcock Medical Center
  • New York
    • Buffalo, New York, United States, 14203
      • The Cystic Fibrosis Center of Western New York
    • Valhalla, New York, United States, 10595
      • New York Medical College at Westchester Medical Center
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27517
      • University of North Carolina at Chapel Hill
  • Ohio
    • Akron, Ohio, United States, 44308
      • Children's Hospital Medical Center of Akron
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center
    • Cleveland, Ohio, United States, 44106
      • Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
    • Columbus, Ohio, United States, 43205
      • Nationwide Children's Hospital
    • Toledo, Ohio, United States, 43606
      • Toledo Children's Hospital
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health Sciences University
  • Pennsylvania
    • Hershey, Pennsylvania, United States, 17033
      • Hershey Medical Center Pennsylvania State University
    • Philadelphia, Pennsylvania, United States, 19104
      • Children's Hospital of Philadelphia
    • Pittsburgh, Pennsylvania, United States, 15224
      • Children's Hospital of Pittsburgh of UPMC
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina
  • Tennessee
    • Memphis, Tennessee, United States, 38103
      • University of Tennessee CF Care and Research Center
    • Nashville, Tennessee, United States, 37232
      • Vanderbilt Children's Hospital
  • Texas
    • Dallas, Texas, United States, 75390
      • University of Texas Southwestern Medical Center
    • Dallas, Texas, United States, 75207
      • University of Texas Southwestern / Children's Health
    • Houston, Texas, United States, 77030
      • Baylor College of Medicine
  • Utah
    • Salt Lake City, Utah, United States, 84113
      • Primary Children's Cystic Fibrosis Center
  • Washington
    • Seattle, Washington, United States, 98195
      • University of Washington Medical Center
    • Seattle, Washington, United States, 98105
      • Seattle Children's Hospital
  • West Virginia
    • Morgantown, West Virginia, United States, 26506
      • West Virginia University - Morgantown
  • Wisconsin
    • Madison, Wisconsin, United States, 53792
      • University of Wisconsin
    • Milwaukee, Wisconsin, United States, 53226
      • Children's Hospital of Wisconsin

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 months and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Cystic Fibrosis Care Centers

Description

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

Additional Inclusion Criteria for CHEC-PKPD Sub-Study:

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)

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
mean change in sweat chloride pre- to post- modulator therapy	mean change in sweat chloride pre- to post- modulator therapy	through study completion, an average of 1 year

Collaborators and Investigators

• Sponsor: Nicole Hamblett
• Collaborators: Cystic Fibrosis Foundation
• Investigators: Principal Investigator: Edith Zemanick, MD, University of Colorado, Denver; Principal Investigator: Michael Konstan, MD, Case Western Reserve University; Principal Investigator: Nicole Mayer-Hamblett, PhD, Seattle Children's Hospital

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (Actual): January 15, 2018
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026

Study Registration Dates

• First Submitted: November 9, 2017
• First Submitted That Met QC Criteria: November 20, 2017
• First Posted (Actual): November 22, 2017

Study Record Updates

• Last Update Posted (Estimated): October 10, 2025
• Last Update Submitted That Met QC Criteria: October 8, 2025
• Last Verified: October 1, 2025

More Information

Terms related to this study

• Keywords: Cystic Fibrosis; CF; CFTR Modulator; Sweat chloride; Sweat
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Respiratory Tract Diseases; Digestive System Diseases; Lung Diseases; Infant, Newborn, Diseases; Pancreatic Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cystic Fibrosis
• Other Study ID Numbers: CHEC-OB-17

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No