What side effects are associated with this type of intervention?

Common treatments for bronchiectasis include antibiotics, bronchodilators, and airway clearance therapies. For treatments similar to CFTR modulation like Trikafta, which is primarily used in cystic fibrosis, known side effects include respiratory symptoms (e.g., cough, nasal congestion), gastrointestinal issues (e.g., diarrhea, abdominal pain), and elevated liver enzymes. Broader side effects for bronchiectasis treatments can include antibiotic resistance, gastrointestinal disturbances, and potential allergic reactions.

What prior FDA approvals exist?

There is no specific mention of prior FDA approvals for CFTR modulation drugs like Trikafta for the treatment of bronchiectasis in the provided context. Trikafta, a combination of elexacaftor, tezacaftor, and ivacaftor, is primarily approved for cystic fibrosis patients with specific CFTR mutations. For bronchiectasis, treatments generally focus on managing symptoms and infections, often using antibiotics, airway clearance techniques, and other supportive therapies.

What other types of research exist?

Promising novel alternatives to Trikafta for Bronchiectasis patients include: 1) Inhaled antibiotics such as tobramycin and aztreonam lysine, which have shown efficacy in managing chronic Pseudomonas aeruginosa infections. 2) Bedaquiline and delamanid, which are being explored for their activity against nontuberculous mycobacteria. 3) Inhaled mannitol, which has demonstrated benefits in improving lung function and mucociliary clearance. These alternatives are based on recent studies and ongoing research in the field.

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CFTR Modulator

Trikafta for Bronchiectasis

Q: What is the mechanism of action of this treatment?

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.

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

Drug Has Already Been Approved

No Placebo-Only Group

Pivotal Trial

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 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline

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

Awards & Highlights

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.

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

2018

Completed Phase 4

~50

0 / 9Eligibility criteria met

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.