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

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ClinicalTrials.gov Identifier: NCT03350828
Recruitment Status : Recruiting
First Posted : November 22, 2017
Last Update Posted : June 12, 2019
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
Nicole Hamblett, Seattle Children's Hospital

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
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.

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Study Type : Observational
Estimated 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 : January 15, 2018
Estimated Primary Completion Date : January 1, 2021
Estimated Study Completion Date : January 1, 2021

Resource links provided by the National Library of Medicine





Primary Outcome Measures :
  1. mean change in sweat chloride pre- to post- modulator therapy [ Time Frame: through study completion, an average of 1 year ]
    mean change in sweat chloride pre- to post- modulator therapy



Information from the National Library of Medicine

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Ages Eligible for Study:   4 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Cystic Fibrosis Care Centers
Criteria

Inclusion Criteria:

  • Written informed consent (and assent when applicable) obtained from subject or subject's legal representative
  • Enrolled in the Cystic Fibrosis Foundation Patient Registry (CFFPR)
  • Male or female ≥ 4 months of age on day of study visit
  • Documentation of a CF diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria:

    • Sweat chloride equal to or greater than 60 milliequivalent (mEq)/liter by quantitative pilocarpine iontophoresis test (QPIT)
    • Two well-characterized mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene
  • Current treatment with a prescribed commercially approved CFTR modulator for at least 3 months prior to enrollment
  • Able to perform the testing and procedures required for this study, as judged by the investigator

Exclusion Criteria:

  • 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
  • Currently enrolled in an investigational trial (including open-label follow-on studies and Expedited Access Pathway (EAP)) of an agent expected to have an impact on sweat chloride (refer to current list provided on study website)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03350828


Contacts
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Contact: Natallia Cameron 206-884-7550 Natallia.Cameron@seattlechildrens.org

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Sponsors and Collaborators
Nicole Hamblett
Cystic Fibrosis Foundation
Investigators
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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

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Responsible Party: Nicole Hamblett, Professor in the Department of Pediatrics in the Division of Pulmonary Medicine, Seattle Children's Hospital
ClinicalTrials.gov Identifier: NCT03350828     History of Changes
Other Study ID Numbers: CHEC-OB-17
First Posted: November 22, 2017    Key Record Dates
Last Update Posted: June 12, 2019
Last Verified: June 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Nicole Hamblett, Seattle Children's Hospital:
CF
Cystic Fibrosis
Sweat
Sweat chloride
CFTR Modulator

Additional relevant MeSH terms:
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Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases