Non-cystic Fibrosis BronchiectasisPhase 4NCT05743946
Trikafta for Patients With Non-cystic Fibrosis Bronchiectasis
A Phase 4 study testing Trikafta in people with Non-cystic Fibrosis Bronchiectasis, run by Emory University at 1 site in United States.
Trial summary
Study participants with non-cystic fibrosis bronchiectasis will be given Trikafta for four weeks. The researchers will monitor clinical endpoints, quality of life, and weight. Additionally, cutaneous punch biopsy material material or blood samples from participants who agree to do this optional test will be collected… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Non-cystic Fibrosis Bronchiectasis
- What is being tested?
- Trikafta
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Apr 2023to 23 Dec 2025
- Phase
- Phase 4
- 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
Who may be able to join
- Provision of signed and dated informed consent form
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Radiologic and other clinical evidence leading to a diagnosis of NCFBE
- 1 CF-causing mutation and/or sweat chloride measurement ≥ 30 mEq/L and < 60 mEq/L
- Able to perform spirometry meeting American Thoracic Society (ATS) criteria for acceptability and repeatability, and FEV1 40-90% predicted
- Clinically stable in the past 4 weeks with no evidence of bronchiectasis exacerbation
Show all 9Show fewer
- Willingness to use at least one form of acceptable birth control including abstinence or condom with spermicide. This will include birth control for at least one month prior to screening and agreement to use such a method during study participation for an additional four weeks after the last administration of Study Drug (for postmenopausal or other women who are without the possibility of becoming pregnant, this requirement may be waived)
- Ability to take Trikafta
- Agreement to adhere to all current medical therapies as designated by the study physician
Who cannot join
- Diagnosis of cystic fibrosis
- Documented history of drug or alcohol abuse within the last year
- Pulmonary exacerbation or changes in therapy for pulmonary disease in the 4 weeks prior to screening
- Listed for lung or liver transplant at the time of screening
- Cirrhosis or elevated liver transaminases > 3 times the upper limit of normal (ULN)
- Pregnant or breastfeeding
Show all 15Show fewer
- Inhibitors or inducers of CYP3A4, including certain herbal medications and grapefruit/grapefruit juice, or other medicines known to negatively influence Trikafta administration
- History of solid organ transplant
- Use of a cardiac pacemaker
- Active therapy for non-tuberculosis mycobacterial infection or any plan to initiate non-tuberculosis mycobacterial therapies during the study period
- Known allergy to Trikafta
- Treatment in the last 6 months with an approved CFTR modulator
- Any other condition that in the opinion of the lead investigators might confound results of the study or pose an additional risk from administering Study Drug
- Treatment with another investigational drug or other intervention within one month prior to enrollment, throughout the duration of study participation, and for an additional four weeks following final drug administration
- Evidence of cataract/lens opacity determined to be clinically significant by an ophthalmologist at or within 3 months prior to the Screening Visit
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Evaluating Trikafta for the Treatment of Patients With Non-cystic Fibrosis Bronchiectasis (NCFBE)
Study participants with non-cystic fibrosis bronchiectasis will be given Trikafta for four weeks. The researchers will monitor clinical endpoints, quality of life, and weight. Additionally, cutaneous punch biopsy material material or blood samples from participants who agree to do this optional test will be collected to test cellular response to Trikafta.
Read the detailed description
Non-cystic fibrosis bronchiectasis (NCFBE) is a clinical syndrome characterized by abnormal dilatation of the airways, airflow obstruction, persistent cough, excessive sputum production and recurrent lung infections. In terms of pathophysiology, airway dilatation and other features are associated with impaired mucociliary clearance and failure to adequately expel bacteria and mucus secretions from the airways. These events contribute to persistent infection, inflammation, and further progressive airway damage, leading to diminished lung function and eventually may cause respiratory failure and death. The pathogenesis of NCFBE is complex, poorly understood, and is likely to vary depending on the underlying etiology and important modifying factors.
Trikafta is approved for patients with cystic fibrosis (CF) carrying at least one copy of the common F508del variant or a number of other cystic fibrosis transmembrane conductance regulator (CFTR) mutations. Trikafta is a combination of three CF drugs, elexacaftor, ivacaftor, and tezacaftor, that helps CFTR proteins work more effectively. Patients with common forms of CF typically exhibit a robust pulmonary benefit from Trikafta within several days to a few weeks of initiating treatment.
NCFBE is clinically and pathologically similar to certain features of cystic fibrosis lung disease. Patients with NCFBE are not approved for Trikafta, and do not have access to the drug. Based on a considerable body of evidence, the researchers believe: 1) cutaneous punch biopsy material and/or blood sample, differentiated to airway epithelium, can be used to identify patients with NCFBE likely to benefit from drugs such as Trikafta, and 2) many patients with NCFBE have a disease likely to exhibit significant clinical improvement when treated with a drug such as Trikafta that activates CFTR-dependent ion transport, although neither of those notions has been adequately tested or proposed previously.
This study is an open-label, single center trial of orally administered elexacaftor, tezacaftor and ivacaftor (Trikafta) that will enroll 30 patients with NCFBE. Study participants will have one known CFTR mutation and/or mildly elevated sweat chloride measurements. In this matter, the study will specifically and prospectively test induced pluripotent stem (iPS) cells taken from patients with NCFBE to determine in vitro thresholds for predicting CFTR rescue in vivo. Using iPS cells differentiated to exhibit a respiratory epithelial phenotype, this study will determine whether the cells can be used to predict FEV1 response among individuals with NCFBE who receive Trikafta.
- Conditions
- Non-cystic Fibrosis Bronchiectasis
- Being tested
- Trikafta
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TrikaftaDrug
Also called elexacaftor, tezacaftor, ivacaftor
Participants will be given elexacaftor 100 mg/tezacaftor 50 mg/ivacaftor 75 mg (two pills once daily in the morning) and ivacaftor 150 mg (once daily in the evening), as the FDA-registered agent, Trikafta. Dose and schedule will be for 28 days, and otherwise identical to what has already been FDA-approved for effective treatment of cystic fibrosis.
| Study group | Role | What they receive |
|---|---|---|
| Trikafta | Experimental | Participants with NCFBE and one known CFTR mutation and/or mildly elevated sweat chloride measurements (i.e., 30-60 mEq/L) receiving Trikafta for four weeks. Receives: Drug: Trikafta |
What is being measured
The primary outcome is the main question the study is designed to answer.
Short Circuit Current Measurements in Monolayers
In vitro responsiveness to Trikafta is tested by determining if iPS cells that are differentiated to airway epithelia and treated with Trikafta display functional correction of CFTR expression. This is assessed by measuring short circuit currents in monolayers.
Measured: Baseline
Western Blot Analysis
In vitro responsiveness to Trikafta is tested by determining if iPS cells that are differentiated to airway epithelia and treated with Trikafta display biological correction of CFTR expression. This is assessed by western blot analysis.
Measured: Baseline
Change in Forced Expiratory Volume in One Second (FEV1)
FEV1 provides a direct measurement of patient health and declines in FEV1 are associated with poor outcomes. FEV1 is measured by spirometry and is the maximum amount of air the participant can blow out in one second. A responder is defined as any subject with an improvement, from baseline, in FEV1 > 5% predicted. FEV1 will also be considered continuously. In this study, if at least 15% of subjects meet the definition of responder, the researchers will view this as initial evidence of a favorable result.
Measured: Baseline, Day 14, Day 28, Day 56
Secondary outcomes (4)
Change in Sweat Chloride Test
Sweat chloride concentrations of less than or equal to 29 milliequivalent per liter (mEq/L) are considered normal, while concentrations of 30-59 mEq/L are considered intermediate. Persons with CF have high levels of chloride in their sweat (concentrations ≥ 60 mEq/L mean that a diagnosis of CF is substantiated). Sweat chloride levels in persons with NCFBE are <60 mEq/L. Studying sweat chloride in persons with NCFBE will provide information regarding the effect of the study intervention using in vivo measures known to indicate CFTR rescue.
Measured: Baseline, Day 14, Day 28, Day 56
Change in Quality of Life-Bronchiectasis (QOL-B) Score
The QOL-B is a 37-item instrument measuring symptoms and health for individuals with NCFBE. The QOL-B includes 8 scales assessing Respiratory Symptoms, Physical, Role, Emotional and Social Functioning, Vitality, Health Perceptions and Treatment Burden. Responses to items are scored from 1 to 4 and scores for each scale are standardized to range from 0 to 100. A total score is not calculated. Higher scores indicate a more favorable health status.
Measured: Baseline, Day 14, Day 28, Day 56
Change in Weight
Weight is measured in kilograms (kg).
Measured: Baseline, Day 14, Day 28, Day 56
Change in Body Mass Index (BMI)
BMI is calculated as the body weight divided by the square of the body height measured in meters (m). BMI is expressed in units of kg/m².
Measured: Baseline, Day 14, Day 28, Day 56
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Feb 2023
- Study start18 Apr 2023
- Primary completion23 Dec 2025
- Study completion23 Dec 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Non-cystic Fibrosis BronchiectasisStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Collaborators
- The Marcus Foundation
- Responsible party
- Principal investigator, Eric Sorscher, Emory University
- Investigators
- Eric Sorscher, MD, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05743946
- Sponsor's protocol ID
- STUDY00004736
- Status last verified
- Jan 2026
- First submitted
- 15 Feb 2023
- Last updated
- 9 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: All of the individual participant data collected during this trial will be made available for sharing, after deidentification.
Publications
Swenson CE, Hunt WR, Manfredi C, Beltran DJ, Hong JS, Davis BR, Suzuki S, Barilla C, Rab A, Chico C, Dangerfield J, Streby A, Barton E, Cox EM, Stecenko AA, Westbrook A, Kapolka R, Sorscher EJ. Evaluating elexacaftor/tezacaftor/ivacaftor (ETI; Trikafta) for treatment of patients with non-cystic fibrosis bronchiectasis (NCFBE): A clinical study protocol. PLoS One. 2025 Feb 14;20(2):e0316721. doi: 10.1371/journal.pone.0316721. eCollection 2025.
PubMed 39951444
Study at a glance
From the registry record, last updated by the sponsor on 9 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluating Trikafta for the Treatment of Patients With Non-cystic Fibrosis Bronchiectasis (NCFBE)
- ClinicalTrials.gov ID
- NCT05743946
- Acronym / sponsor ID
- STUDY00004736
- Conditions
- Non-cystic Fibrosis Bronchiectasis
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Collaborators
- The Marcus Foundation
- Study start
- 18 Apr 2023 (actual)
- Primary completion
- 23 Dec 2025 (actual)
- Study completion
- 23 Dec 2025 (actual)
- First posted
- 24 Feb 2023 (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.
Study participants with non-cystic fibrosis bronchiectasis will be given Trikafta for four weeks. The researchers will monitor clinical endpoints, quality of life, and weight. Additionally, cutaneous punch biopsy material material or blood samples from participants who agree to do this optional test will be collected to test cellular response to Trikafta.
Detailed description
Non-cystic fibrosis bronchiectasis (NCFBE) is a clinical syndrome characterized by abnormal dilatation of the airways, airflow obstruction, persistent cough, excessive sputum production and recurrent lung infections. In terms of pathophysiology, airway dilatation and other features are associated with impaired mucociliary clearance and failure to adequately expel bacteria and mucus secretions from the airways. These events contribute to persistent infection, inflammation, and further progressive airway damage, leading to diminished lung function and eventually may cause respiratory failure and death. The pathogenesis of NCFBE is complex, poorly understood, and is likely to vary depending on the underlying etiology and important modifying factors.
Trikafta is approved for patients with cystic fibrosis (CF) carrying at least one copy of the common F508del variant or a number of other cystic fibrosis transmembrane conductance regulator (CFTR) mutations. Trikafta is a combination of three CF drugs, elexacaftor, ivacaftor, and tezacaftor, that helps CFTR proteins work more effectively. Patients with common forms of CF typically exhibit a robust pulmonary benefit from Trikafta within several days to a few weeks of initiating treatment.
NCFBE is clinically and pathologically similar to certain features of cystic fibrosis lung disease. Patients with NCFBE are not approved for Trikafta, and do not have access to the drug. Based on a considerable body of evidence, the researchers believe: 1) cutaneous punch biopsy material and/or blood sample, differentiated to airway epithelium, can be used to identify patients with NCFBE likely to benefit from drugs such as Trikafta, and 2) many patients with NCFBE have a disease likely to exhibit significant clinical improvement when treated with a drug such as Trikafta that activates CFTR-dependent ion transport, although neither of those notions has been adequately tested or proposed previously.
This study is an open-label, single center trial of orally administered elexacaftor, tezacaftor and ivacaftor (Trikafta) that will enroll 30 patients with NCFBE. Study participants will have one known CFTR mutation and/or mildly elevated sweat chloride measurements. In this matter, the study will specifically and prospectively test induced pluripotent stem (iPS) cells taken from patients with NCFBE to determine in vitro thresholds for predicting CFTR rescue in vivo. Using iPS cells differentiated to exhibit a respiratory epithelial phenotype, this study will determine whether the cells can be used to predict FEV1 response among individuals with NCFBE who receive Trikafta.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Trikafta | Experimental | Drug: TrikaftaParticipants with NCFBE and one known CFTR mutation and/or mildly elevated sweat chloride measurements (i.e., 30-60 mEq/L) receiving Trikafta for four weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Provision of signed and dated informed consent form
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Radiologic and other clinical evidence leading to a diagnosis of NCFBE
- 1 CF-causing mutation and/or sweat chloride measurement ≥ 30 mEq/L and < 60 mEq/L
- Able to perform spirometry meeting American Thoracic Society (ATS) criteria for acceptability and repeatability, and FEV1 40-90% predicted
Key exclusion criteria (15 listed)
- Diagnosis of cystic fibrosis
- Documented history of drug or alcohol abuse within the last year
- Pulmonary exacerbation or changes in therapy for pulmonary disease in the 4 weeks prior to screening
- Listed for lung or liver transplant at the time of screening
- Cirrhosis or elevated liver transaminases > 3 times the upper limit of normal (ULN)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TrikaftaAlso: elexacaftor, tezacaftor, ivacaftorParticipants will be given elexacaftor 100 mg/tezacaftor 50 mg/ivacaftor 75 mg (two pills once daily in the morning) and ivacaftor 150 mg (once daily in the evening), as the FDA-registered agent, Trikafta. Dose and schedule will be for 28 days, and otherwise identical to what has already been FDA-approved for effective treatment of cystic fibrosis. | Drug | Trikafta |
Primary outcome
Short Circuit Current Measurements in Monolayers
Time frame: Baseline
In vitro responsiveness to Trikafta is tested by determining if iPS cells that are differentiated to airway epithelia and treated with Trikafta display functional correction of CFTR expression. This is assessed by measuring short circuit currents in monolayers.
Western Blot Analysis
Time frame: Baseline
In vitro responsiveness to Trikafta is tested by determining if iPS cells that are differentiated to airway epithelia and treated with Trikafta display biological correction of CFTR expression. This is assessed by western blot analysis.
Change in Forced Expiratory Volume in One Second (FEV1)
Time frame: Baseline, Day 14, Day 28, Day 56
FEV1 provides a direct measurement of patient health and declines in FEV1 are associated with poor outcomes. FEV1 is measured by spirometry and is the maximum amount of air the participant can blow out in one second. A responder is defined as any subject with an improvement, from baseline, in FEV1 > 5% predicted. FEV1 will also be considered continuously. In this study, if at least 15% of subjects meet the definition of responder, the researchers will view this as initial evidence of a favorable result.
4 secondary outcomes
Change in Sweat Chloride Test
Time frame: Baseline, Day 14, Day 28, Day 56
Change in Quality of Life-Bronchiectasis (QOL-B) Score
Time frame: Baseline, Day 14, Day 28, Day 56
Change in Weight
Time frame: Baseline, Day 14, Day 28, Day 56
Change in Body Mass Index (BMI)
Time frame: Baseline, Day 14, Day 28, Day 56
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 9 Feb 2026.
- Derived from this study: PubMed 39951444Swenson CE, Hunt WR, Manfredi C, Beltran DJ, Hong JS, Davis BR, Suzuki S, Barilla C, Rab A, Chico C, Dangerfield J, Streby A, Barton E, Cox EM, Stecenko AA, Westbrook A, Kapolka R, Sorscher EJ. Evaluating elexacaftor/tezacaftor/ivacaftor (ETI; Trikafta) for treatment of patients with non-cystic fibrosis bronchiectasis (NCFBE): A clinical study protocol. PLoS One. 2025 Feb 14;20(2):e0316721. doi: 10.1371/journal.pone.0316721. eCollection 2025.
- Individual participant dataSharing planned: Yes. All of the individual participant data collected during this trial will be made available for sharing, after deidentification.
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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.
Eric Sorscher, MD principal investigator
Emory University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Trikafta in people with Non-cystic Fibrosis Bronchiectasis, run by Emory University 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 9 inclusion and 15 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 9 Feb 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.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Short Circuit Current Measurements in Monolayers (time frame: Baseline). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05743946, which also lists 1 linked publication.
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 9 Feb 2026.