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CFTR Modulator

Trikafta for Cystic Fibrosis

Phase 2
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, days 14, 28, 56
Awards & highlights

Study Summary

This trial will test Trikafta on 42 patients who have CFTR mutations that make current CFTR modulator treatments ineffective. The goal is to see if there is a correlation between the in vitro responsiveness of the patients' cells to Trikafta and the in vivo benefit (measured by FEV1) to the patients. If successful, this trial could provide a new tool for using in vitro testing to identify patients most likely to benefit from CFTR modulator therapy.

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.Check my eligibility
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.See study design
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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change in forced expiratory volume in one second (FEV1)
Change in sweat chloride
Response of iPS cells to treatment
Secondary outcome measures
Change in Cystic Fibrosis Questionnaire - Revised (CFQ-R) Score
Change in weight

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.

Find a Location

Who is running the clinical trial?

Cystic Fibrosis FoundationOTHER
189 Previous Clinical Trials
37,168 Total Patients Enrolled
182 Trials studying Cystic Fibrosis
34,231 Patients Enrolled for Cystic Fibrosis
Emory UniversityLead Sponsor
1,640 Previous Clinical Trials
2,560,654 Total Patients Enrolled
20 Trials studying Cystic Fibrosis
1,904 Patients Enrolled for Cystic Fibrosis
National Heart, Lung, and Blood Institute (NHLBI)NIH
3,837 Previous Clinical Trials
47,851,552 Total Patients Enrolled
37 Trials studying Cystic Fibrosis
12,660 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What conditions has Trikafta been found to mitigate?

"Trikafta is often prescribed to individuals who are homozygous for the f508del mutation of cftr gene. It has also been utilised in treating cystic fibrosis, as well as those with ivacaftor-responsive cftr mutations."

Answered by AI

Are there any precedential investigations involving Trikafta?

"At the moment, 21 studies are underway for Trikafta. Of those, 14 of them are in their final phase. With research sites based in Bochum and California, a total number of 1015 locations are conducting trials with this medication."

Answered by AI

Are there any peril associated with the use of Trikafta?

"As Phase 2 trials have data supporting safety but not efficacy, our team at Power gave Trikafta a rating of two on the scale from one to three."

Answered by AI

Is this a groundbreaking medical research study?

"According to the data, Vertex Pharmaceuticals Incorporated have been conducting trials for Trikafta since 2015. Their initial Phase 3 study involved 1044 patients and was successfully concluded. In total, 86 clinical studies have taken place in 109 cities across 23 different countries worldwide; currently 21 are active."

Answered by AI

Is the research team soliciting participants for this study?

"According to information hosted on clinicaltrials.gov, this research is actively recruiting participants since it appeared online in September 2019 and was last updated in March 2022."

Answered by AI

What is the size of the cohort undergoing this research?

"Affirmative, the information on clinicaltrials.gov confirms that this medical research is recruiting participants at present. It was first listed on September 4th 2019 and most recently updated March 25th 2022, with a goal of enrolling 42 people across 3 separate centres."

Answered by AI

Who else is applying?

What state do they live in?
Florida
How old are they?
18 - 65
What site did they apply to?
University of Texas Health Science Center at Houston
What portion of applicants met pre-screening criteria?
Met criteria
Did not meet criteria
Recent research and studies
clinicaltrials.govStudy
Trikafta in Cystic Fibrosis Patients
Eric Sorscher 2019. "Trikafta in Cystic Fibrosis Patients". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03506061.
All > Fibrosis > Cystic Fibrosis
~2 spots leftby Aug 2024
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