Age: 18+
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 4
Recruiting
Sponsor: Emory University
Must not be taking: CYP3A4 inhibitors/inducers
Disqualifiers: Cystic fibrosis, Cirrhosis, Pregnancy, others
No Placebo Group
Prior Safety Data
Breakthrough Therapy
Approved in 4 jurisdictions
Trial Summary
What is the purpose of this trial?This trial will test Trikafta, a medication that helps lung proteins work better, on patients with non-cystic fibrosis bronchiectasis. These patients have lung issues similar to cystic fibrosis but do not respond to typical treatments. The goal is to see if Trikafta can improve their lung health by clearing mucus and bacteria from their airways.
Do I need to stop my current medications to join the trial?
The trial protocol does not specify if you need to stop your current medications, but you must agree to adhere to all current medical therapies as designated by the study physician. Some medications, like those affecting CYP3A4, may not be allowed.
What data supports the effectiveness of the drug Trikafta for bronchiectasis?
Trikafta, a combination of elexacaftor, tezacaftor, and ivacaftor, has been shown to improve lung function and quality of life in patients with cystic fibrosis, a condition that affects the lungs similarly to bronchiectasis. This suggests it might also help improve lung function in bronchiectasis patients.
12345What safety information is available for Trikafta (elexacaftor/tezacaftor/ivacaftor)?
Trikafta, also known as elexacaftor/tezacaftor/ivacaftor, has been associated with some side effects like skin rashes and liver issues in people with cystic fibrosis. Some patients have managed these side effects by adjusting the dose, and in some cases, the rashes resolved without stopping the treatment.
16789How is the drug Trikafta unique for treating bronchiectasis?
Trikafta is unique because it combines three components (elexacaftor, tezacaftor, and ivacaftor) that work together to improve lung function, originally developed for cystic fibrosis, a condition with similar lung issues. This combination targets the underlying cause of the disease, which is different from standard treatments that mainly address symptoms.
135610Eligibility Criteria
This trial is for individuals with non-cystic fibrosis bronchiectasis who can perform spirometry tests and have a certain level of lung function. Participants must not be pregnant, breastfeeding, or have used CFTR modulators in the last 6 months. They should agree to use birth control during the study and remain clinically stable without exacerbations for 4 weeks prior.Inclusion Criteria
I have a CF mutation or my sweat chloride test shows 30-59 mEq/L.
My lung function test meets specific standards and my FEV1 score is between 40-90% of the expected value.
I can take Trikafta without any issues.
+6 more
Exclusion Criteria
I am not taking any medications or consuming products that affect Trikafta.
You are allergic to Trikafta.
I haven't had worsening lung symptoms or changes in my lung treatment in the last 4 weeks.
+11 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment
Participants receive Trikafta for four weeks to assess clinical endpoints, quality of life, and weight.
4 weeks
Weekly visits (in-person)
Follow-up
Participants are monitored for safety and effectiveness after treatment, including changes in FEV1, sweat chloride, and quality of life.
4 weeks
2 visits (in-person)
Participant Groups
The trial is testing Trikafta on patients with non-cystic fibrosis bronchiectasis over four weeks, monitoring clinical outcomes, quality of life, and weight changes. It also involves collecting skin samples to examine cellular responses to the medication.
1Treatment groups
Experimental Treatment
Group I: TrikaftaExperimental Treatment1 Intervention
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.
Trikafta is already approved in United States, European Union, Canada, Australia for the following indications:
🇺🇸 Approved in United States as Trikafta for:
- Cystic fibrosis in patients aged 2 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive based on in vitro data
🇪🇺 Approved in European Union as Trikafta/Kaftrio for:
- Cystic fibrosis in patients aged 2 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive based on in vitro data
🇨🇦 Approved in Canada as Trikafta for:
- Cystic fibrosis in patients aged 2 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive based on in vitro data
🇦🇺 Approved in Australia as Trikafta for:
- Cystic fibrosis in patients aged 2 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive based on in vitro data
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
The Emory ClinicAtlanta, GA
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Who Is Running the Clinical Trial?
Emory UniversityLead Sponsor
The Marcus FoundationCollaborator
The Marcus Foundation, Inc.Collaborator
References
Novel reaction to new cystic fibrosis medication Trikafta. [2021]We present a novel case of an urticaria multiforme-type drug reaction to the new cystic fibrosis medication Trikafta (elexacaftor + tezacaftor + ivacaftor). Equipped with this information, clinicians may be more prepared to counsel and treat patients if they experience similar symptoms after beginning Trikafta.
Effect of highly effective modulator therapy on quality of life in adults with cystic fibrosis. [2021]Elexacaftor/tezacaftor/ivacaftor is a highly effective modulator that improves function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, resulting in improved pulmonary function in patients with cystic fibrosis (CF). We hypothesize that improvements in lung function are associated with improvements in health-related quality of life and sinonasal health. The aim of this study is to measure the effect of elexacaftor/tezacaftor/ivacaftor on patient-reported sinonasal and overall quality of life, and to determine the relationship between changes in these 2 outcome measures.
Elexacaftor/tezacaftor/ivacaftor and gastrointestinal outcomes in cystic fibrosis: Report of promise-GI. [2023]Elexacaftor/tezacaftor/ivacaftor (ETI) improves pulmonary disease in people with cystic fibrosis (PwCF), but its effect on gastrointestinal symptoms, which also affect quality of life, is not clear.
Elexacaftor/Ivacaftor/Tezacaftor: First Approval. [2020]A fixed-dose combination tablet of the cystic fibrosis transmembrane conductance regulator (CFTR) corrector tezacaftor and the CFTR potentiator ivacaftor with the next-generation CFTR corrector elexacaftor (hereafter referred to as elexacaftor/ivacaftor/tezacaftor) [Trikafta™] has been developed by Vertex Pharmaceuticals Inc. to treat patients with the most common cystic fibrosis mutation (F508del). Its use has been associated with statistically significant and/or clinically meaningful improvements in lung function and respiratory-related quality of life compared with comparator regimens (placebo or ivacaftor/tezacaftor) in multinational phase II and III studies, and in October 2019 elexacaftor/ivacaftor/tezacaftor was approved by the US FDA for the treatment of cystic fibrosis in patients aged ≥ 12 years who have ≥ 1 F508del mutation in the CFTR gene. A regulatory assessment for elexacaftor/ivacaftor/tezacaftor as a treatment for cystic fibrosis is underway in the EU. This article summarizes the milestones in the development of elexacaftor/ivacaftor/tezacaftor leading to this first approval for the treatment of cystic fibrosis in patients aged ≥ 12 years who have ≥ 1 F508del mutation in the CFTR gene.
Experience With Elexacaftor/Tezacaftor/Ivacaftor in Patients With Cystic Fibrosis and Advanced Disease. [2023]Elexacaftor/tezacaftor/ivacaftor (ETI) was used through the early access programme in Spain from December 2019 in cystic fibrosis (CF) patients with homozygous or heterozygous F508del mutation with advanced lung disease.
A case of Elexacaftor-Tezacaftor-Ivacaftor induced rash resolving without interruption of treatment. [2022]The triple combination of Elexacaftor-Tezacaftor-Ivacaftor (ELX-TEZ-IVA) has been shown to markedly improve lung function in persons with cystic fibrosis (pwCF). An important adverse effect of the drug is rash, which was reported in clinical trials and highlighted in case reports. Our report demonstrates a similar adverse event with the drug in one of our patients with spontaneous resolution of the rash not necessitating cessation of treatment or desensitization to the drug as were done in other cases. We highlight through our report the heterogeneity of the clinical presentation of the rash when on triple therapy and the need for further studies to understand the immunological mechanism of this adverse event.
If At First You Don't Succeed, Trikafta Again. [2022]Adverse reactions, including severe cutaneous reactions, to cystic fibrosis transmembrane conductance regulator (CFTR) modulators have been described in the literature. Herein we present a drug eruption in response to elexacaftor/tezcaftor/ivacaftor (brand name, Trikafta) in a 7-year-old male with cystic fibrosis, followed by desensitization and successful continuation. A review of the literature outlining similar cases is provided. Attempting to mitigate and manage drug reactions to CFTR modulators is essential because they represent vital and irreplaceable therapies for individuals with cystic fibrosis (CF).
Safety of elexacaftor/tezacaftor/ivacaftor dose reduction: Mechanistic exploration through physiologically based pharmacokinetic modeling and a clinical case series. [2023]Elexacaftor/tezacaftor/ivacaftor (ETI) treatment is associated with significant improvement in lung function in people with cystic fibrosis (pwCF); however, some patients experience adverse effects (AEs) including hepatotoxicity. One potential strategy is dose reduction in ETI with the goal of maintaining therapeutic efficacy while resolving AEs. We report our experience of dose reduction in individuals who experienced AEs following ETI therapy. We provide mechanistic support for ETI dose reduction by exploring predicted lung exposures and underlying pharmacokinetics-pharmacodynamics (PK-PD) relationships.
Effects of elexacaftor/tezacaftor/ivacaftor on liver fibrosis markers in adults with cystic fibrosis. [2023]There are limited studies to date on the effects of elexacaftor/tezacaftor/ivacaftor (E/T/I) on markers of liver fibrosis in adults with cystic fibrosis (CF). This study aims to analyse changes in makers of liver fibrosis before and after initiation of E/T/I in CF adults.
Sustained effectiveness of elexacaftor-tezacaftor-ivacaftor in lung transplant candidates with cystic fibrosis. [2022]Elexacaftor-tezacaftor-ivacaftor induces rapid clinical improvement in patients with cystic fibrosis (CF) and advanced pulmonary disease, often leading to suspend the indication for lung transplantation. Yet no long-term data is available in lung transplant candidates.