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CFTR Modulator

Trikafta for Cystic Fibrosis

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 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

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.

Find a Location

Who is running the clinical trial?

The University of Texas Health Science Center, HoustonOTHER
936 Previous Clinical Trials
335,889 Total Patients Enrolled
Cystic Fibrosis FoundationOTHER
195 Previous Clinical Trials
37,893 Total Patients Enrolled
188 Trials studying Cystic Fibrosis
34,996 Patients Enrolled for Cystic Fibrosis
Emory UniversityLead Sponsor
1,690 Previous Clinical Trials
2,603,351 Total Patients Enrolled
20 Trials studying Cystic Fibrosis
1,904 Patients Enrolled for Cystic Fibrosis

Media Library

Trikafta (CFTR Modulator) Clinical Trial Eligibility Overview. Trial Name: NCT03506061 — Phase 2
Cystic Fibrosis Research Study Groups: Participants With Evidence of Partial Function (sweat chloride < 80 mEq/L or pancreatic sufficiency), Participants who Encode the N1303K Variant
Cystic Fibrosis Clinical Trial 2023: Trikafta Highlights & Side Effects. Trial Name: NCT03506061 — Phase 2
Trikafta (CFTR Modulator) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03506061 — Phase 2
~7 spots leftby Oct 2025