Trikafta for Patients With Non-cystic Fibrosis Bronchiectasis

Prior treatment

• 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
• Inhibitors or inducers of CYP3A4, including certain herbal medications and grapefruit/grapefruit juice, or other medicines known to negatively influence Trikafta administration
• 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

Other illness

• Documented history of drug or alcohol abuse within the last year
• 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
• 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

Lab values

• Cirrhosis or elevated liver transaminases \> 3 times the upper limit of normal (ULN)

Other

• Diagnosis of cystic fibrosis
• Pregnant or breastfeeding
• Treatment in the last 6 months with an approved CFTR modulator

• 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