Trikafta for Patients With Non-cystic Fibrosis Bronchiectasis (NCT05743946) | Clinical Trial Compass
CompletedPhase 4
Trikafta for Patients With Non-cystic Fibrosis Bronchiectasis
United States32 participantsStarted 2023-04-18
Plain-language 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 to test cellular response to Trikafta.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* 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
* 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
Exclusion Criteria:
* 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 (UL…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Short Circuit Current Measurements in Monolayers
Timeframe: Baseline
2
Western Blot Analysis
Timeframe: Baseline
3
Change in Forced Expiratory Volume in One Second (FEV1)