To evaluate the clinical mechanisms of action in lung and extrapulmonary systems of VX-661 (tezacaftor; TEZ) in combination with ivacaftor (IVA) (TEZ/IVA) in participants with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
34
Tezacaftor/Ivacaftor FDC
Unnamed facility
Birmingham, Alabama, United States
Unnamed facility
Indianapolis, Indiana, United States
Unnamed facility
Baltimore, Maryland, United States
Unnamed facility
Chapel Hill, North Carolina, United States
Absolute Change From Baseline in Mucociliary Clearance (MCC) at Day 28
MCC was assessed using an imaging technique that enables the tracking of mucus within the airways. MCC was expressed as the percentage of whole-lung clearance through 60 minutes at Baseline and Day 28.
Time frame: Baseline, Day 28
Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28
Percent predicted FEV1 is the ratio of FEV1 to the predicted FEV1, expressed as a percentage. FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Time frame: Baseline, Day 28
Absolute Change From Baseline in Small-bowel Area Under the Curve (AUC) Over 1-minute Mean pH Increments at Day 29
Absolute change from Baseline in small bowel AUC over 1-minute mean pH increments through 30 minutes at Day 29 was assessed.
Time frame: Baseline, Day 29
Absolute Change From Baseline in Sweat Chloride at Day 29
Time frame: Baseline, Day 29
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Baseline up to Day 57
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Unnamed facility
Cincinnati, Ohio, United States
Unnamed facility
Toledo, Ohio, United States
Unnamed facility
Pittsburgh, Pennsylvania, United States