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Observational Trial of the Longitudinal Effects of CFTR Modulator Drugs

Sponsor
National Jewish Health (Other)
Overall Status
Recruiting
CT.gov ID
NCT04353752
Collaborator
Cystic Fibrosis Foundation (Other)
80
Enrollment
1
Location
74.5
Anticipated Duration (Months)
1.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

CF patients enrolled in this protocol will be recruited from patients followed by the Adult CF Program at National Jewish Health. Patients will be selected based on planned use of a CFTR modulator by their primary physician. No patient will be started on (or will switch) CFTR modulator agents for the purpose of the study. After enrollment, biological samples may be collected at two different time points prior to treatment initiation. One set of samples will be collected at baseline health prior to CFTR modulator initiation or change. A second set of samples will be obtained in subjects at the time of acute pulmonary exacerbation, if one occurs prior to CFTR modulator initiation or change. Post CFTR modulator initiation study assessments will be obtained at least one month after starting treatment and continue up to 2 times a year (including during pulmonary exacerbations), in order to document longitudinal effects of therapies and changes in inflammatory biology over time. At the time of each biological sampling, blood, sputum sample, urine, and a quality of life assessment will be acquired in all enrolled subjects. In addition to demographic data, clinical data, such as quantitative microbiology and simple spirometry will be recorded at the time of sample collection.

Condition or Disease Intervention/Treatment Phase

    Study Design

    Study Type:
    Observational
    Anticipated Enrollment :
    80 participants
    Observational Model:
    Cohort
    Time Perspective:
    Prospective
    Official Title:
    The Natural History of Cystic Fibrosis in the Era of Next Gen Combination Modulator Agents: Novel Treatment Targets in an Evolving Disease
    Actual Study Start Date :
    Sep 16, 2019
    Anticipated Primary Completion Date :
    Dec 1, 2023
    Anticipated Study Completion Date :
    Dec 1, 2025

    Outcome Measures

    Primary Outcome Measures

    1. Change in blood inflammatory markers [Before initiation/change of CFTR modulator and after, on average 6 months apart]

      Changes in host inflammation as measured in peripheral blood memory T cells

    2. Change in sputum microbiology [Before initiation/change of CFTR modulator and after, on average 6 months apart]

      Changes in the bacterial burden in sputum as measured by bacterial RNA

    Secondary Outcome Measures

    1. Changes in lung function [Before initiation/change of CFTR modulator and after, on average 6 months apart]

      Changes in lung function as measured by FEV1, % predicted

    2. Changes in urine inflammatory marker [Before initiation/change of CFTR modulator and after, on average 6 months apart]

      Changes in host inflammation as measured by urinary neutrophil gelatinase-associated lipocalin (NGAL)

    3. Changes in quality of life [Before initiation/change of CFTR modulator and after, on average 6 months apart]

      Changes in quality of life as measured by Cystic Fibrosis Questionnaire - Revised (CFQ-R)

    4. Changes in hospitalizations [Before initiation/change of CFTR modulator and after, on average 6 months apart]

      Changes in frequency/number of hospitalizations for treatment of pulmonary exacerbation

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    • Documented diagnosis of CF with an eligible mutation for modulator treatment.

    • Age 18 years old or older.

    • Patient is starting a CFTR modulator as part of their clinical care or switching CFTR modulator therapy agents, pending FDA approval.

    • Ability to perform reproducible Pulmonary Function Tests.

    • Willingness to comply with study procedure and willingness to provide written consent.

    Exclusion Criteria:

    • Presence of a condition or abnormality that, in the opinion of the Principal Investigator (PI), would compromise the safety of the patient or the quality of the data.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 National Jewish Health Denver Colorado United States 80206

    Sponsors and Collaborators

    • National Jewish Health
    • Cystic Fibrosis Foundation

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    National Jewish Health
    ClinicalTrials.gov Identifier:
    NCT04353752
    Other Study ID Numbers:
    • SAAVED19AO-PG
    First Posted:
    Apr 20, 2020
    Last Update Posted:
    Sep 10, 2020
    Last Verified:
    Sep 1, 2020
    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
    Product Manufactured in and Exported from the U.S.:
    No
    Additional relevant MeSH terms:
    Cystic Fibrosis
    Fibrosis

    Study Results

    No Results Posted as of Sep 10, 2020