A Phase 3 interventional study of TEZ/IVA and IVA in Cystic Fibrosis, sponsored by Vertex Pharmaceuticals Incorporated. Completed at 55 sites in 11 countries. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2024-04-19.
Sponsored by Vertex Pharmaceuticals Incorporated · Phase 3, Interventional, and Treatment
This study evaluates the long-term safety and tolerability of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 years and older, homozygous or heterozygous for the F508del mutation.
1,605 studies on the registry are indexed under Cystic Fibrosis; 196 are open to participants now.
This study's enrollment of 130 is above the median of 36 across 1,051 interventional studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →Vertex Pharmaceuticals Incorporated is the lead sponsor of 249 studies on the registry; 20 are open to participants now.
Of its 106 completed or terminated interventional studies of FDA-regulated products, 53 (50%) have results posted.
Counted across the registry records on this site, refreshed daily.
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Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria may apply.
Part A: Participants weighing less than (\<)40 kilograms (kg) at Day 1 received tezacaftor (TEZ) 50 milligrams (mg) once daily (qd)/ivacaftor (IVA) 75 mg every 12 hours (q12h) and the participants weighing greater than or equals to (\>=) 40 kg at Day 1 received TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 96 weeks. Doses were adjusted upward for changes in body weight and/or age. Part B: Participants weighing \<30 kg at Day 1 received TEZ 50 mg qd/IVA 75 mg q12h and the participants weighing \>=30 kg at Day 1 received TEZ 100 mg qd/IVA 150 mg q12h in the treatment period up to 192 weeks. Doses were adjusted upward for changes in body weight and/or age.
Drug: TEZ/IVA · Drug: IVA
Drug pages: Tezacaftor · Ivacaftor
Fixed-dose combination tablet for oral administration.
Also known as: VX-661/VX-770, tezacaftor/ivacaftor
Tablet for oral administration.
Also known as: VX-770, ivacaftor
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: Day 1 up to Week 100
Part A: Absolute Change in Lung Clearance Index2.5 (LCI2.5) for 115/116 FAS (TEZ/IVA Group)
The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.
Time frame: From Parent Study 115 Baseline at Week 96 (Study 116)
Part A: Absolute Change in LCI2.5 for 113B/116 LCI FAS
The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.
Time frame: From Parent Study 113B Baseline at Week 96 (Study 116)
Part A: Absolute Change in Sweat Chloride (SwCl) for 115/116 FAS (TEZ/IVA Group)
Sweat samples were collected using an approved collection device.
Time frame: From Parent Study 115 Baseline at Week 96 (Study 116)
Part A: Absolute Change in SwCl for 113B/116 FAS
Sweat samples were collected using an approved collection device.
Time frame: From Parent Study 113B Baseline at Week 96 (Study 116)
Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 115/116 FAS (TEZ/IVA Group)
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.
Time frame: From Parent Study 115 Baseline at Week 96 (Study 116)
Part A: Absolute Change in CFQ-R Respiratory Domain Score for 113B/116 FAS
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.
Time frame: From Parent Study 113B Baseline at Week 96 (Study 116)
Part A: Absolute Change in Body Mass Index (BMI) for 115/116 FAS (TEZ/IVA Group)
BMI was defined as weight in kilograms (kg) divided by squared height in meters (m\^2).
Time frame: From Parent Study 115 Baseline at Week 96 (Study 116)
Part A: Absolute Change in BMI for 113B/116 FAS
BMI was defined as weight in kg divided by m\^2.
Time frame: From Parent Study 113B Baseline at Week 96 (Study 116)
Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Day 1 up to Week 192
The study was conducted in 2 parts, Part A and Part B. Participants from Part A also participated in Part B. The Participant flow was planned to be presented for the overall treatment arm.
| Milestone | TEZ/IVA |
|---|---|
| Started | 130 |
| 113b/116 fas | 64 |
| 113b/116 lci fas | 30 |
| 115/116 fas | 66 |
| 115/116 fas (tez/iva group) | 53 |
| Completed | 69 |
| Not completed | 61 |
| Withdrew: Adverse event | 2 |
| Withdrew: Withdrawal of consent (not due to ae) | 2 |
| Withdrew: Commercial drug is available for participant | 56 |
| Withdrew: Other | 1 |
| Milestone | TEZ/IVA |
|---|---|
| Started | 62 |
| 113b/116 fas | 9 |
| 115/116 fas | 53 |
| Completed | 3 |
| Not completed | 59 |
| Withdrew: Adverse event | 1 |
| Withdrew: Withdrawal of consent (not due to ae) | 1 |
| Withdrew: Commercial drug is available for participant | 56 |
| Withdrew: Other | 1 |
| Participants | Part A: TEZ/IVA |
|---|---|
| Participants With TEAEs | 129 |
| Participants With SAEs | 31 |
The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.
| Index | Part A: TEZ/IVA |
|---|---|
| Part A: Absolute Change in Lung Clearance Index2.5 (LCI2.5) for 115/116 FAS (TEZ/IVA Group) | -0.95 (-1.38 to -0.52) |
The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.
| Index | Part A: TEZ/IVA |
|---|---|
| Part A: Absolute Change in LCI2.5 for 113B/116 LCI FAS | -2.04 ± 1.73 |
Sweat samples were collected using an approved collection device.
| millimole per liter (mmol/L) | TEZ/IVA |
|---|---|
| Part A: Absolute Change in Sweat Chloride (SwCl) for 115/116 FAS (TEZ/IVA Group) | -13.8 (-17.7 to -9.9) |
Sweat samples were collected using an approved collection device.
| mmol/L | Part A: TEZ/IVA |
|---|---|
| Part A: Absolute Change in SwCl for 113B/116 FAS | -16.2 (-21.9 to -10.5) |
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.
| units on a scale | Part A: TEZ/IVA |
|---|---|
| Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 115/116 FAS (TEZ/IVA Group) | 6.4 (3.5 to 9.3) |
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.
| units on a scale | Part A: TEZ/IVA |
|---|---|
| Part A: Absolute Change in CFQ-R Respiratory Domain Score for 113B/116 FAS | 6.0 (1.1 to 10.8) |
BMI was defined as weight in kilograms (kg) divided by squared height in meters (m\^2).
| kilogram per meter square (kg/m^2) | Part A: TEZ/IVA |
|---|---|
| Part A: Absolute Change in Body Mass Index (BMI) for 115/116 FAS (TEZ/IVA Group) | 1.25 (1.00 to 1.49) |
BMI was defined as weight in kg divided by m\^2.
| kg/m^2 | Part A: TEZ/IVA |
|---|---|
| Part A: Absolute Change in BMI for 113B/116 FAS | 1.19 (0.74 to 1.64) |
| Participants | TEZ/IVA |
|---|---|
| Participants with TEAEs | 53 |
| Participants with SAEs | 8 |
Collected over Day 1 Through Safety Follow-up Visit (up to Week 100 for Part A, and up to Week 192 for Part B). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Part A: TEZ/IVA | 0/130 (0%) | 31/130 (23.8%) | 128/130 (98.5%) |
| Part B: TEZ/IVA | 0/62 (0%) | 8/62 (12.9%) | 49/62 (79%) |
| Event | Part A: TEZ/IVA | Part B: TEZ/IVA |
|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosisInfections and infestations | 15/130 | 1/62 |
| Nasal polypsRespiratory, thoracic and mediastinal disorders | 1/130 | 2/62 |
| Bacterial test positiveInvestigations | 3/130 | 1/62 |
| Abdominal painGastrointestinal disorders | 2/130 | 1/62 |
| GastroenteritisInfections and infestations | 1/130 | 1/62 |
| Septic shockInfections and infestations | 0/130 | 1/62 |
| Upper respiratory tract infectionInfections and infestations | 0/130 | 1/62 |
| Procedural haemorrhageInjury, poisoning and procedural complications | 0/130 | 1/62 |
| Pseudomonas test positiveInvestigations | 0/130 | 1/62 |
| Adenoidal hypertrophyRespiratory, thoracic and mediastinal disorders | 0/130 | 1/62 |
| Event | Part A: TEZ/IVA | Part B: TEZ/IVA |
|---|---|---|
| CoughRespiratory, thoracic and mediastinal disorders | 73/130 | 15/62 |
| Infective pulmonary exacerbation of cystic fibrosisInfections and infestations | 57/130 | 10/62 |
| COVID-19Infections and infestations | 0/130 | 15/62 |
| Upper respiratory tract infectionInfections and infestations | 31/130 | 10/62 |
| PyrexiaGeneral disorders | 26/130 | 3/62 |
| NasopharyngitisInfections and infestations | 24/130 | 10/62 |
| Nasal congestionRespiratory, thoracic and mediastinal disorders | 24/130 | 2/62 |
| Oropharyngeal painRespiratory, thoracic and mediastinal disorders | 24/130 | 6/62 |
| Productive coughRespiratory, thoracic and mediastinal disorders | 22/130 | 6/62 |
| VomitingGastrointestinal disorders | 21/130 | 5/62 |
Baseline is defined as the most recent non-missing measurement (scheduled or unscheduled) collected before the first dose of TEZ/IVA in either the parent study or Study 116. Baseline data is presented for participants who received at least 1 dose of study drug in this study.
| Age, Continuous(years) | TEZ/IVA |
|---|---|
| Part A | 8.3 ± 1.7 |
| Part B | 8.3 ± 1.7 |
| Sex: Female, Male(Participants) | TEZ/IVA |
|---|---|
| Part A — Female | 67 |
| Part A — Male | 63 |
| Part B — Female | 35 |
| Part B — Male | 27 |
| Race/Ethnicity, Customized(Participants) | TEZ/IVA |
|---|---|
| Part A — Hispanic or Latino | 4 |
| Part A — Not Hispanic or Latino | 119 |
| Part A — Not collected per local regulations | 7 |
| Part B — Hispanic or Latino | 3 |
| Part B — Not Hispanic or Latino | 52 |
| Part B — Not collected per local regulations | 7 |
| Race/Ethnicity, Customized(Participants) | TEZ/IVA |
|---|---|
| Part A — White | 125 |
| Part A — Black or African American | 1 |
| Part A — Asian | 1 |
| Part A — Not collected per local regulations | 2 |
| Part A — Other | 1 |
| Part B — White | 59 |
| Part B — Black or African American | 1 |
| Part B — Asian | 0 |
| Part B — Not collected per local regulations | 2 |
| Part B — Other | 0 |
Documents are hosted by the registry — open the source record to download them.
Ran from Apr 2018 to primary completion in Oct 2020, 30 months.
Results were first posted in Nov 2021, 1 year after primary completion.
Of 58 industry-sponsored phase 3 interventional studies under Cystic Fibrosis, half reached primary completion within 25 months of starting (the median), counting those still running; a quarter within 14 and three quarters within 36. This study's 30 months is above that.
Counting every sponsor, half of 66 studies reached it within 26 months; a quarter within 16 and three quarters within 39.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Mar 2024. You cannot join it, but the record below documents what was studied.
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Studies of the drugs tested here: Tezacaftor · Ivacaftor
Vertex Pharmaceuticals Incorporated →