RECRUITING

Trikafta for Patients With Non-cystic Fibrosis Bronchiectasis

Conditions

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

Evaluating Trikafta for the Treatment of Patients With Non-cystic Fibrosis Bronchiectasis (NCFBE)

Quick Facts

Study Start:2023-04-18

Study Completion:2025-07

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05743946

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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	* 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

Inclusion Criteria

Exclusion 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

* 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

Contacts and Locations

Study Contact

Eric Sorscher, MD

CONTACT

205-612-1327

esorscher@emory.edu

Randy Hunt, MD

CONTACT

317-289-1197

randy.hunt@emory.edu

Principal Investigator

Eric Sorscher, MD

PRINCIPAL_INVESTIGATOR

Emory University

Study Locations (Sites)

The Emory Clinic

Atlanta, Georgia, 30322

United States

Collaborators and Investigators

Sponsor: Emory University

Eric Sorscher, MD, PRINCIPAL_INVESTIGATOR, Emory University

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-04-18

Study Completion Date2025-07

Study Record Updates

Study Start Date2023-04-18

Study Completion Date2025-07

Terms related to this study

Additional Relevant MeSH Terms

Non-cystic Fibrosis Bronchiectasis