A Phase 2 Study to Evaluate Effects of VX-661/Ivacaftor on Lung and Extrapulmonary Systems in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
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|ClinicalTrials.gov Identifier: NCT02508207|
Recruitment Status : Completed
First Posted : July 24, 2015
Results First Posted : July 27, 2018
Last Update Posted : July 19, 2021
|Condition or disease||Intervention/treatment||Phase|
|Cystic Fibrosis||Drug: Tezacaftor/Ivacaftor Drug: Ivacaftor Drug: Tezacaftor/Ivacaftor matching placebo Drug: Ivacaftor matching placebo||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||34 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Phase 2, Randomized, Double-Blind, Placebo Controlled, Parallel-Group, Exploratory Study to Evaluate Effects of VX-661 in Combination With Ivacaftor on Lung and Extrapulmonary Systems in Subjects Aged 18 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation|
|Study Start Date :||February 2016|
|Actual Primary Completion Date :||June 2017|
|Actual Study Completion Date :||June 2017|
Placebo Comparator: Placebo
Participants received placebo matched to tezacaftor (TEZ)/ivacaftor (IVA) fixed dose combination (FDC) tablet orally once daily in the morning followed by placebo matched to IVA tablet orally once daily in the evening for 29 days.
Drug: Tezacaftor/Ivacaftor matching placebo
Drug: Ivacaftor matching placebo
Participants received 100 milligram (mg) TEZ/150 mg IVA FDC tablet orally once daily in the morning followed by 150 mg IVA tablet orally once daily in the evening for 29 days.
- Absolute Change From Baseline in Mucociliary Clearance (MCC) at Day 28 [ Time Frame: Baseline, 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.
- Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28 [ Time Frame: Baseline, 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.
- Absolute Change From Baseline in Small-bowel Area Under the Curve (AUC) Over 1-minute Mean pH Increments at Day 29 [ Time Frame: Baseline, Day 29 ]Absolute change from Baseline in small bowel AUC over 1-minute mean pH increments through 30 minutes at Day 29 was assessed.
- 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 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02508207
|United States, Alabama|
|Birmingham, Alabama, United States|
|United States, Indiana|
|Indianapolis, Indiana, United States|
|United States, Maryland|
|Baltimore, Maryland, United States|
|United States, North Carolina|
|Chapel Hill, North Carolina, United States|
|United States, Ohio|
|Cincinnati, Ohio, United States|
|Toledo, Ohio, United States|
|United States, Pennsylvania|
|Pittsburgh, Pennsylvania, United States|