A Phase 3 Study of Tezacaftor (VX-661) in Combination With Ivacaftor (VX-770) in Subjects Aged 12 Years and Older With Cystic Fibrosis (CF), Who Have One F508del-CFTR Mutation and a Second Mutation That Has Been Demonstrated to be Clinically Responsive to Ivacaftor
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|ClinicalTrials.gov Identifier: NCT02412111|
Recruitment Status : Completed
First Posted : April 8, 2015
Results First Posted : January 8, 2019
Last Update Posted : January 8, 2019
|Condition or disease||Intervention/treatment||Phase|
|Cystic Fibrosis||Drug: Ivacaftor Drug: Tezacaftor/Ivacaftor||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||156 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Phase 3, Randomized, Double-Blind, Ivacaftor-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation and a Second CFTR Allele With a Gating Defect That Is Clinically Demonstrated to be Ivacaftor Responsive|
|Actual Study Start Date :||June 2015|
|Actual Primary Completion Date :||September 2017|
|Actual Study Completion Date :||September 2017|
Experimental: Ivacaftor (Run-in Period)
Ivacaftor 150 milligram (mg) tablet orally every 12 hours for 4 weeks.
Other Name: VX-770
Experimental: VX-661 + Ivacaftor (Active comparator period)
VX-661 100 mg and ivacaftor 150 mg fixed-dose combination tablet orally once daily in the morning and ivacaftor 150 mg tablet orally once daily in the evening for 8 weeks.
Other Name: VX-770
Other Name: VX-661/VX-770
Active Comparator: Ivacaftor monotherapy (Active comparator period)
Ivacaftor 150 mg tablet orally every 12 hours as monotherapy for 8 weeks.
Other Name: VX-770
- Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 8 [ Time Frame: Baseline, Through Week 8 ]FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
- Relative Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 8 [ Time Frame: Baseline, Through Week 8 ]FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
- Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score From Baseline Through Week 8 [ Time Frame: Baseline, Through Week 8 ]The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
- Absolute Change From Baseline in Sweat Chloride Through Week 8 [ Time Frame: Baseline, Through Week 8 ]Sweat samples were collected using an approved collection device.
- Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Baseline up to Week 16 ]
- Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolites (M1-VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA) [ Time Frame: Predose on Week -2 for Run-in period; Pre-dose on Week 2 for Active comparator period ]
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): NCT02412111
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