A Study to Evaluate Safety, Efficacy, and Tolerability of TEZ/IVA in Orkambi® (Lumacaftor/Ivacaftor) -Experienced Subjects With Cystic Fibrosis (CF)
|ClinicalTrials.gov Identifier: NCT03150719|
Recruitment Status : Completed
First Posted : May 12, 2017
Results First Posted : September 12, 2019
Last Update Posted : September 12, 2019
|Condition or disease||Intervention/treatment||Phase|
|Cystic Fibrosis||Drug: Tezacaftor/Ivacaftor Drug: Ivacaftor Drug: Placebo||Phase 3|
Expanded Access : An investigational treatment associated with this study has been approved for sale to the public. More info ...
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||98 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||Phase 3b, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Assess the Safety, Efficacy, and Tolerability of Tezacaftor/Ivacaftor (TEZ/IVA) in an Orkambi-experienced Population Who Are Homozygous for the F508del CFTR Mutation|
|Actual Study Start Date :||May 24, 2017|
|Actual Primary Completion Date :||August 9, 2018|
|Actual Study Completion Date :||August 9, 2018|
Placebo Comparator: Placebo
Participants received placebo matched to TEZ/IVA fixed-dose combination tablet orally once daily in the morning followed by placebo matched to IVA tablet orally once daily in the evening for 56 days.
Placebo matched to TEZ/IVA fixed-dose combination tablet.
Placebo matched to IVA tablet.
Participants received TEZ 100 milligram (mg)/IVA 150 mg fixed-dose combination tablet orally once daily in the morning and IVA 150 mg tablet orally once daily in the evening for 56 days.
TEZ 100 mg/IVA 150 mg fixed-dose combination tablet.
Other Name: TEZ/IVA; VX-661/VX-770
IVA 150 mg tablet.
Other Name: VX-770; IVA
- Incidence of Respiratory Adverse Events of Special Interest (RAESIs) [ Time Frame: Day 1 up to Day 84 ]RAESIs included chest discomfort, dyspnea (shortness of breath), respiration abnormal (chest tightness), asthma, bronchial hyperreactivity, bronchospasm, and wheezing.
- Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Average of Day 28 and Day 56 Measurements [ Time Frame: Baseline, Day 28 and Day 56 ]FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
- Relative Change From Baseline in ppFEV1 at Average of Day 28 and Day 56 Measurements [ Time Frame: Baseline, Day 28 and Day 56 ]FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
- Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Average of Day 28 and Day 56 Measurements [ Time Frame: Baseline, Day 28 and Day 56 ]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.
- Tolerability as Assessed by Number of Participants Who Discontinued Treatment [ Time Frame: Day 1 through Day 56 ]
- Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Day 1 up to Day 84 ]
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03150719