What side effects are associated with this type of intervention?

CFTR modulator therapies, such as Trikafta, are commonly used in the treatment of cystic fibrosis. Known side effects of these treatments include respiratory symptoms like increased cough and nasal congestion, gastrointestinal issues such as diarrhea and abdominal pain, and elevated liver enzymes indicating potential liver function impact. Other side effects may include rash, headache, and dizziness. It is important for patients to discuss these potential side effects with their healthcare provider to manage and monitor any adverse reactions effectively.

What prior FDA approvals exist?

The FDA has approved several CFTR modulator therapies for the treatment of cystic fibrosis, targeting the underlying genetic mutations. Notable approvals include ivacaftor (Kalydeco), which was the first CFTR modulator approved for patients with specific gating mutations. Lumacaftor/ivacaftor (Orkambi) and tezacaftor/ivacaftor (Symdeko) followed, targeting patients with the F508del mutation. Trikafta, a combination of elexacaftor, tezacaftor, and ivacaftor, represents a significant advancement, approved for patients with at least one F508del mutation, expanding treatment options for a broader patient population.

What other types of research exist?

Promising novel alternatives to Trikafta for Cystic Fibrosis patients include the ongoing evaluation of elexacaftor, tezacaftor, and ivacaftor combinations for different CFTR mutations, as well as other emerging CFTR modulators and correctors being tested in clinical trials. Additionally, therapies targeting specific bacterial infections and inflammation in CF patients, such as inhaled antibiotics and anti-inflammatory agents, are also under investigation and may provide complementary benefits.

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CFTR Modulator

Trikafta for Cystic Fibrosis

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

CFTR modulator therapies, such as Trikafta, work by targeting the defective CFTR protein caused by mutations in the CFTR gene. These treatments enhance the function of the CFTR protein, which regulates the movement of salt and water in and out of cells. By improving CFTR function, these therapies help reduce the thick, sticky mucus buildup in the lungs, improving lung function and reducing the frequency of pulmonary exacerbations. This is vital for Cystic Fibrosis patients as it significantly enhances respiratory health and overall quality of life.

Phase 2

Waitlist Available

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline

Awards & highlights

No Placebo-Only Group

Summary

This trial will test Trikafta on cystic fibrosis patients with rare mutations not currently approved for this treatment. The study aims to see if lab-grown cells from these patients can predict how well they will respond to Trikafta. This could help more patients with rare mutations get effective treatments. Trikafta is a combination therapy consisting of elexacaftor, tezacaftor, and ivacaftor, which has shown effectiveness in treating certain CFTR mutations.

Who is the study for?

This trial is for cystic fibrosis patients aged 12 or older who don't have the F508del mutation but may have partial function mutations or N1303K. They should be clinically stable, able to perform spirometry tests, and not on certain CFTR modulators. Participants must agree to use birth control and adhere to their current medical therapies.

What is being tested?

The study is testing Trikafta in a small group of cystic fibrosis patients with specific genetic profiles over four weeks. Researchers will monitor lung function and sweat chloride levels, as well as test patient-derived iPS cells in the lab to predict clinical response.

What are the potential side effects?

While specific side effects are not listed here, Trikafta can cause liver issues, rash, flu-like symptoms among others. Patients with a history of drug allergies or significant alcohol abuse are excluded from this trial.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, days 7, 14, 28, 56

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, days 7, 14, 28, 56

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, days 7, 14, 28, 56 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)

Change in sweat chloride

Response of iPS cells to treatment

Secondary study objectives

Change in Cystic Fibrosis Questionnaire - Revised (CFQ-R) Score

Change in weight

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Participants who Encode the N1303K VariantExperimental Treatment1 Intervention

Participants with CF who encode the N1303K variant will receive Trikafta for 28 days.

Group II: Participants With Evidence of Partial Function (sweat chloride < 80 mEq/L or pancreatic sufficiency)Experimental Treatment1 Intervention

Participants with CF with evidence of partial function (sweat chloride \< 80 milliequivalents per liter (mEq/L) or pancreatic sufficiency) will receive Trikafta for 28 days.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Trikafta

2019

Completed Phase 2

~50

Do I qualify?Apply below to find out

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

CFTR modulator therapies, such as Trikafta, work by targeting the defective CFTR protein caused by mutations in the CFTR gene. These treatments enhance the function of the CFTR protein, which regulates the movement of salt and water in and out of cells. By improving CFTR function, these therapies help reduce the thick, sticky mucus buildup in the lungs, improving lung function and reducing the frequency of pulmonary exacerbations. This is vital for Cystic Fibrosis patients as it significantly enhances respiratory health and overall quality of life.