Cystic FibrosisNCT04353752
Observational Trial of the Longitudinal Effects of CFTR Modulator Drugs
An observational study of Cystic Fibrosis, run by National Jewish Health at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- How many people
- 58 participants
- Where
- 1 siteUnited States
- Study dates
- 16 Sep 2019to 26 Jul 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: The Natural History of Cystic Fibrosis in the Era of Next Gen Combination Modulator Agents: Novel Treatment Targets in an Evolving Disease
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
- Medical subject headings
- Cystic Fibrosis
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in blood inflammatory markers
Changes in host inflammation as measured in peripheral blood memory T cells
Measured: 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
Measured: Before initiation/change of CFTR modulator and after, on average 6 months apart
Secondary outcomes (4)
Changes in lung function
Changes in lung function as measured by FEV1, % predicted
Measured: Before initiation/change of CFTR modulator and after, on average 6 months apart
Changes in urine inflammatory marker
Changes in host inflammation as measured by urinary neutrophil gelatinase-associated lipocalin (NGAL)
Measured: Before initiation/change of CFTR modulator and after, on average 6 months apart
Changes in quality of life
Changes in quality of life as measured by Cystic Fibrosis Questionnaire - Revised (CFQ-R)
Measured: Before initiation/change of CFTR modulator and after, on average 6 months apart
Changes in hospitalizations
Changes in frequency/number of hospitalizations for treatment of pulmonary exacerbation
Measured: Before initiation/change of CFTR modulator and after, on average 6 months apart
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Apr 2020
- Study start16 Sep 2019
- Primary completion12 Jan 2021
- Study completion26 Jul 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 58 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Jewish Health
- Sponsor type
- University, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04353752
- Sponsor's protocol ID
- SAAVED19AO-PG
- Status last verified
- Apr 2026
- First submitted
- 20 Mar 2020
- Last updated
- 13 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Natural History of Cystic Fibrosis in the Era of Next Gen Combination Modulator Agents: Novel Treatment Targets in an Evolving Disease
- ClinicalTrials.gov ID
- NCT04353752
- Acronym / sponsor ID
- SAAVED19AO-PG
- Conditions
- Cystic Fibrosis
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 58 participants (actual)
- Lead sponsor
- National Jewish HealthUniversity, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Study start
- 16 Sep 2019 (actual)
- Primary completion
- 12 Jan 2021 (actual)
- Study completion
- 26 Jul 2023 (actual)
- First posted
- 20 Apr 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (1 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Change in blood inflammatory markers
Time frame: 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
Time frame: 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
4 secondary outcomes
Changes in lung function
Time frame: Before initiation/change of CFTR modulator and after, on average 6 months apart
Changes in urine inflammatory marker
Time frame: Before initiation/change of CFTR modulator and after, on average 6 months apart
Changes in quality of life
Time frame: Before initiation/change of CFTR modulator and after, on average 6 months apart
Changes in hospitalizations
Time frame: Before initiation/change of CFTR modulator and after, on average 6 months apart
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Apr 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by National Jewish Health at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in blood inflammatory markers (time frame: Before initiation/change of CFTR modulator and after, on average 6 months apart). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Apr 2026.