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CFTR Modulator

Trikafta for Bronchiectasis

Phase 4
Recruiting
Led By Eric Sorscher, MD
Research Sponsored by Emory University
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Presence of 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
Must not have
Inhibitors or inducers of CYP3A4, including certain herbal medications and grapefruit/grapefruit juice, or other medicines known to negatively influence Trikafta administration
Active therapy for non-tuberculosis mycobacterial infection or any plan to initiate non-tuberculosis mycobacterial therapies during the study period
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, day 14, day 28, day 56
Awards & highlights

Summary

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.

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
While specific side effects are not listed here, potential risks may include allergic reactions to Trikafta or interactions with other medications. Side effects could range from mild symptoms like headaches or digestive issues to more serious conditions affecting liver function.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have a CF mutation or my sweat chloride test shows 30-59 mEq/L.
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My lung function test meets specific standards and my FEV1 score is between 40-90% of the expected value.
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I can take Trikafta without any issues.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I am not taking any medications or consuming products that affect Trikafta.
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I am not receiving or planning to start treatment for non-tuberculosis mycobacterial infections during the study.
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My liver is not working well or my liver tests are high.
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I am on the waiting list for a lung or liver transplant.
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I have had a solid organ transplant.
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I have been diagnosed with cystic fibrosis.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change in Forced Expiratory Volume in One Second (FEV1).
Short Circuit Current Measurements in Monolayers
Western Blot Analysis
Secondary study objectives
Change in Body Mass Index (BMI)
Change in Quality of Life-Bronchiectasis (QOL-B) Score
Change in Sweat Chloride Test
+1 more

Trial Design

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.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Trikafta
2019
Completed Phase 2
~50

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatments for Bronchiectasis include antibiotics to manage infections, bronchodilators to open airways, and mucolytics to thin mucus. Trikafta, a CFTR modulator, works by improving the function of the CFTR protein, which is defective in cystic fibrosis. This modulation helps in reducing mucus viscosity and improving mucociliary clearance. For Bronchiectasis patients, especially those with CFTR mutations, such treatments can significantly improve lung function, reduce exacerbations, and enhance overall quality of life by addressing the underlying cause of mucus buildup and recurrent infections.
Current and Future Treatments in Primary Ciliary Dyskinesia.

Find a Location

Who is running the clinical trial?

Emory UniversityLead Sponsor
1,688 Previous Clinical Trials
2,602,885 Total Patients Enrolled
The Marcus Foundation, Inc.UNKNOWN
Eric Sorscher, MDPrincipal InvestigatorEmory University
1 Previous Clinical Trials
42 Total Patients Enrolled

Media Library

Trikafta (CFTR Modulator) Clinical Trial Eligibility Overview. Trial Name: NCT05743946 — Phase 4
Bronchiectasis Research Study Groups: Trikafta
Bronchiectasis Clinical Trial 2023: Trikafta Highlights & Side Effects. Trial Name: NCT05743946 — Phase 4
Trikafta (CFTR Modulator) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05743946 — Phase 4
~9 spots leftby Jul 2025