Observational Trial of the Longitudinal Effects of CFTR Modulator Drugs

Active, not recruiting

• Conditions: Cystic Fibrosis

• Registration Number: NCT04353752
• Lead Sponsor: National Jewish Health

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2019-09-16
• Study First Submitted: 2020-03-20
• Study First Submitted QC: 2020-04-16
• Study First Posted: 2020-04-20
• Status Verified: 2022-09
• Last Update Submitted: 2022-09-28
• Last Update Posted: 2022-09-30
• Primary Completion: 2023-12
• Study Completion: 2025-12

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 80

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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
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

Name	Time	Method
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
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)
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
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)

Trial Locations

Locations (1)

National Jewish Health; 🇺🇸 Denver, Colorado, United States