Observational Trial of the Longitudinal Effects of CFTR Modulator Drugs

NCT04353752 | Completed

• 1 other identifier: SAAVED19AO-PG
• Study Type: observational
• Target: 58
• Locations: 1 country
• Sites: 1

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.

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (2)

• Change in blood inflammatory markers

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

  Before initiation/change of CFTR modulator and after, on average 6 months apart

• Change in sputum microbiology

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

  Before initiation/change of CFTR modulator and after, on average 6 months apart

Secondary Outcomes (4)

• Changes in lung function

  Before initiation/change of CFTR modulator and after, on average 6 months apart

• Changes in urine inflammatory marker

  Before initiation/change of CFTR modulator and after, on average 6 months apart

• Changes in quality of life

  Before initiation/change of CFTR modulator and after, on average 6 months apart

• Changes in hospitalizations

  Before initiation/change of CFTR modulator and after, on average 6 months apart

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Subjects enrolled in this protocol will be recruited from patients followed by the Adult CF Program at National Jewish Health. The investigators project enrolling 80 subjects with diagnosed CF, 18 years of age or older, with eligible mutations for CFTR modulator therapies. Concomitant use of systemic steroids will be allowed, and typical co-infections or co-morbidities will not result in exclusion.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Cystic Fibrosis Foundation: collaborator
• National Jewish Health: lead

Study Sites (1)

National Jewish Health

Denver, Colorado, 80206, United States

Location

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 20, 2020
• First Posted: April 20, 2020
• Study Start: September 16, 2019
• Primary Completion: January 12, 2021
• Study Completion: July 26, 2023
• Last Updated: April 13, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)