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

Sponsor
Nicole Hamblett (Other)
Overall Status
Active, not recruiting
CT.gov ID
NCT03350828
Collaborator
Cystic Fibrosis Foundation (Other)
5,000
Enrollment
52
Locations
71.5
Anticipated Duration (Months)
96.2
Patients Per Site
1.3
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease Intervention/Treatment Phase

    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 Design

    Study Type:
    Observational
    Anticipated Enrollment :
    5000 participants
    Observational Model:
    Cohort
    Time Perspective:
    Prospective
    Official Title:
    The CHEC-SC Cohort Study: CHaractErizing CFTR Modulated Changes in Sweat Chloride and Their Association With Clinical Outcomes
    Actual Study Start Date :
    Jan 15, 2018
    Anticipated Primary Completion Date :
    Dec 31, 2023
    Anticipated Study Completion Date :
    Dec 31, 2023

    Outcome Measures

    Primary Outcome Measures

    1. mean change in sweat chloride pre- to post- modulator therapy [through study completion, an average of 1 year]

      mean change in sweat chloride pre- to post- modulator therapy

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    4 Months and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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)

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 The Children's Hospital Alabama, University of Alabama at Birmingham Birmingham Alabama United States 35233
    2 University of Arkansas for Medical Sciences Little Rock Arkansas United States 72205
    3 University of California San Diego La Jolla California United States 92093
    4 Childrens Hospital Los Angeles Los Angeles California United States 90027
    5 Children's Hospital Colorado Aurora Colorado United States 80045
    6 National Jewish Health Denver Colorado United States 80206
    7 Yale University School of Medicine New Haven Connecticut United States 06520
    8 University of Florida Gainesville Florida United States 32610
    9 The Nemours Children's Clinic - Orlando Orlando Florida United States 32827
    10 Nemours Children's Clinic - Pensacola Pensacola Florida United States 32514
    11 Children's Healthcare of Atlanta and Emory University Atlanta Georgia United States 30322
    12 Emory University Atlanta Georgia United States 30327
    13 Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois United States 60611
    14 University of Chicago Chicago Illinois United States 60637
    15 Riley Hospital for Children Indianapolis Indiana United States 46202
    16 University of Kansas Medical Center Kansas City Kansas United States 66160
    17 University of Kentucky Lexington Kentucky United States 40506
    18 John Hopkins Hospital Baltimore Maryland United States 21287
    19 Massachusetts General Hospital Boston Massachusetts United States 02114
    20 Boston Children's Hospital Boston Massachusetts United States 02115
    21 University of Michigan, Michigan Medicine Ann Arbor Michigan United States 48109
    22 Children's Hospital of Michigan Detroit Michigan United States 48201
    23 Helen DeVos Children's Hospital Grand Rapids Michigan United States 49503
    24 The Minnesota Cystic Fibrosis Center Minneapolis Minnesota United States 55455
    25 Children's Mercy Kansas City Kansas City Missouri United States 64108
    26 SSM Health Cardinal Glennon Children's Hospital Saint Louis Missouri United States 63104
    27 St. Louis Children's Hospital Saint Louis Missouri United States 63110
    28 Dartmouth Hitchcock Medical Center Lebanon New Hampshire United States 03756
    29 The Cystic Fibrosis Center of Western New York Buffalo New York United States 14203
    30 New York Medical College at Westchester Medical Center Valhalla New York United States 10595
    31 University of North Carolina at Chapel Hill Chapel Hill North Carolina United States 27517
    32 Children's Hospital Medical Center of Akron Akron Ohio United States 44308
    33 Cincinnati Children's Hospital Medical Center Cincinnati Ohio United States 45229
    34 Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center Cleveland Ohio United States 44106
    35 Nationwide Children's Hospital Columbus Ohio United States 43205
    36 Toledo Children's Hospital Toledo Ohio United States 43606
    37 Oregon Health Sciences University Portland Oregon United States 97239
    38 Hershey Medical Center Pennsylvania State University Hershey Pennsylvania United States 17033
    39 Children's Hospital of Philadelphia Philadelphia Pennsylvania United States 19104
    40 Children's Hospital of Pittsburgh of UPMC Pittsburgh Pennsylvania United States 15224
    41 Medical University of South Carolina Charleston South Carolina United States 29425
    42 University of Tennessee CF Care and Research Center Memphis Tennessee United States 38103
    43 Vanderbilt Children's Hospital Nashville Tennessee United States 37232
    44 University of Texas Southwestern / Children's Health Dallas Texas United States 75207
    45 University of Texas Southwestern Medical Center Dallas Texas United States 75390
    46 Baylor College of Medicine Houston Texas United States 77030
    47 Primary Children's Cystic Fibrosis Center Salt Lake City Utah United States 84113
    48 Seattle Children's Hospital Seattle Washington United States 98105
    49 University of Washington Medical Center Seattle Washington United States 98195
    50 West Virginia University - Morgantown Morgantown West Virginia United States 26506
    51 University of Wisconsin Madison Wisconsin United States 53792
    52 Children's Hospital of Wisconsin Milwaukee Wisconsin United States 53226

    Sponsors and Collaborators

    • Nicole Hamblett
    • 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

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Nicole Hamblett, Professor in the Department of Pediatrics in the Division of Pulmonary Medicine, Seattle Children's Hospital
    ClinicalTrials.gov Identifier:
    NCT03350828
    Other Study ID Numbers:
    • CHEC-OB-17
    First Posted:
    Nov 22, 2017
    Last Update Posted:
    Jun 21, 2022
    Last Verified:
    Jun 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Nicole Hamblett, Professor in the Department of Pediatrics in the Division of Pulmonary Medicine, Seattle Children's Hospital
    CF
    Cystic Fibrosis
    Sweat
    Sweat chloride
    CFTR Modulator
    Additional relevant MeSH terms:
    Cystic Fibrosis
    Fibrosis

    Study Results

    No Results Posted as of Jun 21, 2022