A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Subjects With an F508del CFTR Mutation
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ClinicalTrials.gov Identifier: NCT03537651 |
Recruitment Status :
Active, not recruiting
First Posted : May 25, 2018
Results First Posted : November 26, 2021
Last Update Posted : November 26, 2021
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Condition or disease | Intervention/treatment | Phase |
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Cystic Fibrosis | Drug: TEZ/IVA Drug: IVA | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 130 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Tezacaftor in Combination With Ivacaftor in Subjects With Cystic Fibrosis Aged 6 Years and Older, Homozygous or Heterozygous for the F508del-CFTR Mutation |
Actual Study Start Date : | April 25, 2018 |
Actual Primary Completion Date : | October 28, 2020 |
Estimated Study Completion Date : | September 2022 |

Arm | Intervention/treatment |
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Experimental: TEZ/IVA
TEZ 50 mg once daily (qd)/IVA 75 mg every 12 hours (q12h) or TEZ 100 mg qd/IVA 150 mg q12h based on body weight for participants aged 6 through 11 years at enrollment and TEZ 100 mg qd/IVA 150 mg q12h for participants aged >=12 years at enrollment. Doses were adjusted upward for changes in body weight and/or age.
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Drug: TEZ/IVA
Fixed-dose combination tablet for oral administration.
Other Names:
Drug: IVA Tablet for oral administration.
Other Names:
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- Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Part A: Day 1 up to Week 100 ]
- Part A: Absolute Change in Lung Clearance Index2.5 (LCI2.5) for 115/116 FAS (TEZ/IVA Group) [ Time Frame: From Parent Study 115 Baseline at Week 96 (Study 116) ]LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
- Part A: Absolute Change in LCI2.5 for 113B/116 LCI FAS [ Time Frame: From Parent Study 113B Baseline at Week 96 (Study 116) ]LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
- Part A: Absolute Change in Sweat Chloride for 115/116 FAS (TEZ/IVA Group) [ Time Frame: From Parent Study 115 Baseline at Week 96 (Study 116) ]Sweat samples were collected using an approved collection device.
- Part A: Absolute Change in SwCl for 113B/116 FAS [ Time Frame: From Parent Study 113B Baseline at Week 96 (Study 116) ]Sweat samples were collected using an approved collection device.
- Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 115/116 FAS (TEZ/IVA Group) [ Time Frame: From Parent Study 115 Baseline at Week 96 (Study 116) ]The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with CF. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
- Part A: Absolute Change in CFQ-R Respiratory Domain Score for 113B/116 FAS [ Time Frame: From Parent Study 113B Baseline at Week 96 (Study 116) ]The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with CF. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
- Part A: Absolute Change in Body Mass Index (BMI) for 115/116 FAS (TEZ/IVA Group) [ Time Frame: From Parent Study 115 Baseline at Week 96 (Study 116) ]BMI was defined as weight in kilograms (kg) divided by squared height in meters (m^2).
- Part A: Absolute Change in BMI for 113B/116 FAS [ Time Frame: From Parent Study 113B Baseline at Week 96 (Study 116) ]BMI was defined as weight in kg divided by m^2.

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Ages Eligible for Study: | 6 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Completed the Week 24 Visit in Study 113 Part B or the Week 8 Visit in Study 115.
- Eligible CFTR Mutation.
Exclusion Criteria:
- Pregnant and nursing females.
- History of poor compliance with study drug and/or procedures in a previous study as deemed by the investigator.
- Ongoing participation in another study with investigational drug.
Other protocol defined Inclusion/Exclusion criteria may apply.

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): NCT03537651

Documents provided by Vertex Pharmaceuticals Incorporated:
Responsible Party: | Vertex Pharmaceuticals Incorporated |
ClinicalTrials.gov Identifier: | NCT03537651 |
Other Study ID Numbers: |
VX17-661-116 2017-002968-40 ( EudraCT Number ) |
First Posted: | May 25, 2018 Key Record Dates |
Results First Posted: | November 26, 2021 |
Last Update Posted: | November 26, 2021 |
Last Verified: | October 2021 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Cystic Fibrosis Fibrosis Pathologic Processes Pancreatic Diseases Digestive System Diseases Lung Diseases Respiratory Tract Diseases |
Genetic Diseases, Inborn Infant, Newborn, Diseases Ivacaftor Chloride Channel Agonists Membrane Transport Modulators Molecular Mechanisms of Pharmacological Action |