Status:
COMPLETED
A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function
Lead Sponsor:
Nicole Hamblett
Collaborating Sponsors:
Cystic Fibrosis Foundation
Conditions:
Cystic Fibrosis
Eligibility:
All Genders
12+ years
Brief Summary
This is a prospective, multi-center observational study. The study is designed to measure the clinical effectiveness of elexacaftor, tezacaftor and ivacaftor (ETI) triple combination therapy in people...
Detailed Description
Cystic fibrosis (CF) is an autosomal recessive genetic disorder caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein. In people with CF, this...
Eligibility Criteria
Inclusion
- All genders within the age limit of the FDA approved indication for elexacaftor, tezacaftor and ivacaftor triple combination therapy (TCT) at Day 1.
- Diagnosis of CF.
- CFTR mutations consistent with the FDA approved indication for elexacaftor, tezacaftor and ivacaftor triple combination therapy (TCT).
- Physician intent to prescribe elexacaftor, tezacaftor and ivacaftor triple combination therapy (TCT).
- Willing to fast for 8 hours prior to all study visits (for subjects on overnight enteric tube feedings, willing to hold the feeding for at least 8 hours).
- Able to perform the testing and procedures required for this study, as judged by the investigator.
- Enrolled in the Cystic Fibrosis Foundation Patient Registry.
- Clinically stable with no significant changes in health status within the 14 days prior to Visit 1.
Exclusion
- Use of any TCT within the 180 days prior to Visit 1.
- Any acute use of antibiotics (oral, inhaled or IV) or systemic corticosteroids within the 2 weeks prior to Visit 1 for lower respiratory tract symptoms.
- Initiation of any new chronic therapy (e.g., ibuprofen, Pulmozyme®, hypertonic saline, azithromycin, inhaled tobramycin, Cayston®, Kalydeco, Orkambi®, Symdeko®) within the 4 weeks prior to Visit 1.
- Use of an investigational agent within the 28 days prior to Visit 1.
- Use of chronic oral corticosteroids (equivalent to 10 mg. or more per day of prednisone) within the 28 days prior to Visit 1.
- Treatment for nontuberculous mycobacterial (NTM) infection, consisting of ≥ two antibiotics (oral, IV, and/or inhaled) within the 28 days prior to Visit 1.
- History of lung or liver transplantation, or listing for organ transplantation.
Key Trial Info
Start Date :
October 22 2019
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
December 23 2024
Estimated Enrollment :
490 Patients enrolled
Trial Details
Trial ID
NCT04038047
Start Date
October 22 2019
End Date
December 23 2024
Last Update
January 28 2025
Active Locations (56)
Enter a location and click search to find clinical trials sorted by distance.
1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35294
2
Providence Alaska Medical Center
Anchorage, Alaska, United States, 99508
3
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States, 72205
4
Stanford University Medical Center
Palo Alto, California, United States, 94025