A Study to Evaluate Safety, Efficacy, and Tolerability of TEZ/IVA in Orkambi® (Lumacaftor/Ivacaftor) -Experienced Subjects With Cystic Fibrosis (CF)
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|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 ]
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): NCT03150719