Using Trikafta to treat patients with non-cystic fibrosis bronchiectasis
Evaluating Trikafta for the Treatment of Patients With Non-cystic Fibrosis Bronchiectasis (NCFBE)
This study is testing if the medication Trikafta can help improve the health and quality of life for people with non-cystic fibrosis bronchiectasis.
Quick facts
| Phase | Phase 4 |
|---|---|
| Study type | Interventional |
| Enrollment | 30 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Emory University Academic / other |
| Locations | 1 site (Atlanta, Georgia) |
| Trial ID | NCT05743946 on ClinicalTrials.gov |
What this trial studies
This study investigates the effects of Trikafta, a medication typically used for cystic fibrosis, on patients diagnosed with non-cystic fibrosis bronchiectasis (NCFBE). Participants will receive Trikafta for four weeks, during which researchers will monitor clinical endpoints, quality of life, and weight changes. Additionally, optional cutaneous punch biopsy or blood samples will be collected to assess cellular responses to the treatment. The study aims to explore whether Trikafta can provide benefits to patients with NCFBE, a condition that shares similarities with cystic fibrosis.
Who should consider this trial
Good fit: Ideal candidates include individuals diagnosed with non-cystic fibrosis bronchiectasis who meet specific clinical and spirometry criteria.
Not a fit: Patients with severe bronchiectasis exacerbations or those not meeting the eligibility criteria may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could improve lung function and quality of life for patients with non-cystic fibrosis bronchiectasis.
How similar studies have performed: While Trikafta is primarily approved for cystic fibrosis, this approach is novel for non-cystic fibrosis bronchiectasis and has not been extensively tested in similar studies.
Eligibility criteria
Show full inclusion / exclusion criteria
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 * 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 (ULN) * Pregnant or breastfeeding * 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 * 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
Where this trial is running
Atlanta, Georgia
- The Emory Clinic — Atlanta, Georgia, United States (Recruiting)
Study contacts
- Principal investigator: Eric Sorscher, MD — Emory University
- Study coordinator: Eric Sorscher, MD
- Email: esorscher@emory.edu
- Phone: 205-612-1327
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.