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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 belowSelect...
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:Select...
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 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline
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 moreTrial 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.
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
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am not taking any medications or consuming products that affect Trikafta.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.You are allergic to Trikafta.I haven't had worsening lung symptoms or changes in my lung treatment in the last 4 weeks.I am not receiving or planning to start treatment for non-tuberculosis mycobacterial infections during the study.My liver is not working well or my liver tests are high.I am willing and able to follow the study rules and attend all required visits.You have a record of using drugs or drinking too much alcohol in the past year.I have taken CFTR modulator medication in the past 6 months.I am on the waiting list for a lung or liver transplant.I have had a solid organ transplant.I have been diagnosed with cystic fibrosis.An eye doctor found significant cataract in my eye within the last 3 months.You have been diagnosed with non-cystic fibrosis bronchiectasis based on X-rays or other medical tests.My lung condition has been stable for the last 4 weeks without worsening.
Research Study Groups:
This trial has the following groups:- Group 1: Trikafta
Awards:
This trial has 3 awards, including:- Drug Has Already Been Approved - The FDA has already approved this drug, and is just seeking more data.
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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