Observational Trial of the Longitudinal Effects of CFTR Modulator Drugs

Cystic Fibrosis Clinical Trial

Official title:

The Natural History of Cystic Fibrosis in the Era of Next Gen Combination Modulator Agents: Novel Treatment Targets in an Evolving Disease

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04353752
• Other study ID #: SAAVED19AO-PG
• Status: Active, not recruiting
• Start date: September 16, 2019
• Est. completion date: December 2025

Study information

• Verified date: September 2022
• Source: National Jewish Health
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 80
• Est. completion date: December 2025
• Est. primary completion date: December 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Locations

Country	Name	City	State
United States	National Jewish Health	Denver	Colorado

Sponsors (2)

Lead Sponsor	Collaborator
National Jewish Health	Cystic Fibrosis Foundation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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	Changes in lung function	Changes in lung function as measured by FEV1, % predicted	Before initiation/change of CFTR modulator and after, on average 6 months apart
Secondary	Changes in urine inflammatory marker	Changes in host inflammation as measured by urinary neutrophil gelatinase-associated lipocalin (NGAL)	Before initiation/change of CFTR modulator and after, on average 6 months apart
Secondary	Changes in quality of life	Changes in quality of life as measured by Cystic Fibrosis Questionnaire - Revised (CFQ-R)	Before initiation/change of CFTR modulator and after, on average 6 months apart
Secondary	Changes in hospitalizations	Changes in frequency/number of hospitalizations for treatment of pulmonary exacerbation	Before initiation/change of CFTR modulator and after, on average 6 months apart