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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-002968-40 | EudraCT Number |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| TEZ/IVA | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| TEZ/IVA | Drug | Fixed-dose combination tablet for oral administration. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Day 1 up to Week 100 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria may apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35233 | United States | ||
| Providence Alaska Medical Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37983082 | Derived | Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4. | |
| 35190292 | Derived |
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This study was conducted in cystic fibrosis (CF) participants aged 6 years or older who participated in parent studies VX15-661-113 Part B (Study 113B; NCT02953314) or VX16-661-115 (Study 115; NCT03559062). Eligible participants from parent studies were enrolled in Study 116.
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.
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| ID | Title | Description |
|---|---|---|
| FG000 | TEZ/IVA | 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 upto192 weeks. Doses were adjusted upward for changes in body weight and/or age. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Part A (Up to 96 Weeks) |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Nov 8, 2019 | Oct 21, 2021 |
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| IVA | Drug | Tablet for oral administration. |
|
|
| 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. | 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. | 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. | 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. | 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. | 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). | 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. | 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) | Day 1 up to Week 192 |
| Anchorage |
| Alaska |
| 99508 |
| United States |
| Arkansas Children's Hospital | Little Rock | Arkansas | 72202 | United States |
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | United States |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | United States |
| Nemours/ Alfred I. duPont Hospital for Children | Wilmington | Delaware | 19803 | United States |
| Johns Hopkins All Children's Hospital Outpatient Care Center | St. Petersburg | Florida | 33701 | United States |
| Center for Advanced Pediatrics | Atlanta | Georgia | 30329 | United States |
| St. Luke's CF Center of Idaho | Boise | Idaho | 83702 | United States |
| Riley Hospital for Children Indiana University Health | Indianapolis | Indiana | 46202 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| Children's Hospital & Clinics of Minnesota | Minneapolis | Minnesota | 55404 | United States |
| The Children's Mercy Hospital | Kansas City | Missouri | 64108 | United States |
| Dartmouth Hitchcock Medical Center | Manchester | New Hampshire | 03756 | United States |
| UBMD Pediatrics/ CF Center of Western New York | Buffalo | New York | 14203 | United States |
| Columbia University Medical Center | New York | New York | 10032 | United States |
| SUNY Upstate Medical University | Syracuse | New York | 13202 | United States |
| Wake Forest Baptist Health | Winston-Salem | North Carolina | 27157 | United States |
| Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center | Cleveland | Ohio | 44106 | United States |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15224 | United States |
| Medical University of South Carolina (MUSC) | Charleston | South Carolina | 29425 | United States |
| Sanford Children's Speciality Clinic | Sioux Falls | South Dakota | 57105 | United States |
| Austin Children's Chest Associates | Austin | Texas | 78723 | United States |
| Cook Children's Medical Center | Fort Worth | Texas | 76104 | United States |
| Baylor College of Medicine | Houston | Texas | 77030 | United States |
| Children's Hospital of The King's Daughters | Norfolk | Virginia | 23507 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| Children's Hospital of Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
| Perth Children's Hospital | Nedlands | Australia |
| John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital | New Lambton | Australia |
| Lady Cilento Children's Hospital | South Brisbane | Australia |
| The Children's Hospital at Westmead | Westmead | Australia |
| Universitair Ziekenhuis Brussel - Campus Jette | Brussels | Belgium |
| Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg | Leuven | Belgium |
| British Columbia's Children's Hospital | Vancouver | British Columbia | Canada |
| The Hospital for Sick Children | Toronto | Ontario | Canada |
| McGill University Health Centre, Glen Site, Montreal Children's Hospital | Montreal | Quebec | Canada |
| Juliane Marie Center, Rigshopitalet | Copenhagen | Denmark |
| Groupe Hospitaler Pellegrin, CHU De Bordeaux | Bordeaux | France |
| Hopital Necker, Enfants Malades | Paris | France |
| Universitaetsklinkum Koeln, CF-Studienzentrum | Cologne | Germany |
| Universitätsklinikum Essen | Essen | Germany |
| Clinic of J.W. Goethe University | Frankfurt | Germany |
| Justus-Leibig-Universitat Zentrum fur Kinderheilkunde und Jugendmedizin | Giessen | Germany |
| Medizinische Hochschule Hannover | Hanover | Germany |
| Universitaetsklinikum Jena, Mukoviszidose-Zentrum | Jena | Germany |
| Universitaetsklinikum Tuebingen Klinik fuer Kinder- und Jugendmedizin | Tübingen | Germany |
| Our Lady's Children's Hospital | Dublin | Ireland |
| University Hospital Limerick | Limerick | Ireland |
| Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY | Łomianki | Poland |
| Inselspital - Universitaetsspital Bern | Bern | Switzerland |
| Kinderspital Zuerich | Zurich | Switzerland |
| Leeds General Infirmary | Leeds | United Kingdom |
| Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital | London | United Kingdom |
| Southampton General Hospital | Southampton | United Kingdom |
| Sawicki GS, Chilvers M, McNamara J, Naehrlich L, Saunders C, Sermet-Gaudelus I, Wainwright CE, Ahluwalia N, Campbell D, Harris RS, Paz-Diaz H, Shih JL, Davies JC. A Phase 3, open-label, 96-week trial to study the safety, tolerability, and efficacy of tezacaftor/ivacaftor in children >/= 6 years of age homozygous for F508del or heterozygous for F508del and a residual function CFTR variant. J Cyst Fibros. 2022 Jul;21(4):675-683. doi: 10.1016/j.jcf.2022.02.003. Epub 2022 Feb 18. |
| 33331662 | Derived | Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3. |
| 113B/116 FAS | All enrolled participants from parent Study 113B who received at least 1 dose of TEZ/IVA in Study 116 and had an eligible genotype. |
|
| 113B/116 LCI FAS | All enrolled participants who participated in the LCI sub study in parent Study 113B and received at least 1 dose of TEZ/IVA in Study 116 and had an eligible genotype. |
|
| 115/116 FAS | All enrolled participants who were randomized to either TEZ/IVA, IVA or placebo treatment group in parent Study 115 and received at least 1 dose of TEZ/IVA in Study 116 and had an eligible genotype. |
|
| 115/116 FAS (TEZ/IVA Group) | All enrolled participants who were randomized to the TEZ/IVA treatment group in parent Study 115 and received at least 1 dose of TEZ/IVA in Study 116 and had an eligible genotype. |
|
| COMPLETED |
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| NOT COMPLETED |
|
|
| Part B (Up to 192 Weeks) |
|
|
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.
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| ID | Title | Description |
|---|---|---|
| BG000 | TEZ/IVA | Part A: Participants weighing <40 kg at Day 1 received TEZ 50 mg qd/ IVA 75 mg q12h and the participants weighing >= 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study. | Mean | Standard Deviation | years |
| ||||||||||||||||
| Sex: Female, Male | The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study. | Count of Participants | Participants | No |
| ||||||||||||||||
| Race/Ethnicity, Customized | The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study. | Count of Participants | Participants | No |
| ||||||||||||||||
| Race/Ethnicity, Customized | The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study. | Count of Participants | Participants | No |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Safety Set included all participants who were enrolled and received at least 1 dose of TEZ/IVA in Part A of Study 116. | Posted | Count of Participants | Participants | No | Day 1 up to Week 100 |
|
|
| |||||||||||||||||||||||||||||||||
| Secondary | 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. | 115/116 Full analysis set (FAS) (TEZ/IVA group) included all enrolled participants who were randomized to the TEZ/IVA treatment group in parent Study 115 and received at least 1 dose of TEZ/IVA in Study 116 and had an eligible genotype. As pre-specified in the SAP, model-based efficacy analysis for participants from parent Study 115 was planned only for the TEZ/IVA treatment group. | Posted | Least Squares Mean | 95% Confidence Interval | Index | From Parent Study 115 Baseline at Week 96 (Study 116) |
|
| |||||||||||||||||||||||||||||||||
| Secondary | 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. | 113B/116 LCI FAS included all enrolled participants who participated in the LCI sub study in parent Study 113B and received at least 1 dose of TEZ/IVA in Study 116 and had an eligible genotype. As pre-specified in the SAP, only descriptive summary statistics were planned to be reported for the 113B/116 LCI FAS. | Posted | Mean | Standard Deviation | Index | From Parent Study 113B Baseline at Week 96 (Study 116) |
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| Secondary | Part A: Absolute Change in Sweat Chloride (SwCl) for 115/116 FAS (TEZ/IVA Group) | Sweat samples were collected using an approved collection device. | 115/116 FAS (TEZ/IVA group). As pre-specified in the SAP, model-based efficacy analysis for participants from parent Study 115 was planned only for the TEZ/IVA treatment group. | Posted | Least Squares Mean | 95% Confidence Interval | millimole per liter (mmol/L) | From Parent Study 115 Baseline at Week 96 (Study 116) |
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| |||||||||||||||||||||||||||||||||
| Secondary | Part A: Absolute Change in SwCl for 113B/116 FAS | Sweat samples were collected using an approved collection device. | 113B/116 FAS included all enrolled participants from parent Study 113B who received at least 1 dose of TEZ/IVA in Study 116 and had an eligible genotype. | Posted | Least Squares Mean | 95% Confidence Interval | mmol/L | From Parent Study 113B Baseline at Week 96 (Study 116) |
|
| |||||||||||||||||||||||||||||||||
| Secondary | 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. | 115/116 FAS (TEZ/IVA group). As pre-specified in the SAP, model-based efficacy analysis for participants from parent Study 115 was planned only for the TEZ/IVA treatment group. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | From Parent Study 115 Baseline at Week 96 (Study 116) |
|
| |||||||||||||||||||||||||||||||||
| Secondary | 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. | 113B/116 FAS. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | From Parent Study 113B Baseline at Week 96 (Study 116) |
|
| |||||||||||||||||||||||||||||||||
| Secondary | 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). | 115/116 FAS (TEZ/IVA group). As pre-specified in the SAP, model-based efficacy analysis for participants from parent Study 115 was planned only for the TEZ/IVA treatment group. | Posted | Least Squares Mean | 95% Confidence Interval | kilogram per meter square (kg/m^2) | From Parent Study 115 Baseline at Week 96 (Study 116) |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Part A: Absolute Change in BMI for 113B/116 FAS | BMI was defined as weight in kg divided by m^2. | 113B/116 FAS. | Posted | Least Squares Mean | 95% Confidence Interval | kg/m^2 | From Parent Study 113B Baseline at Week 96 (Study 116) |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Safety Set included all participants who were enrolled and received at least 1 dose of TEZ/IVA in Part B of Study 116. | Posted | Count of Participants | Participants | No | Day 1 up to Week 192 |
|
|
Day 1 Through Safety Follow-up Visit (up to Week 100 for Part A, and up to Week 192 for Part B)
Safety Set included all participants who were enrolled and received at least 1 dose of TEZ/IVA in Study 116.MedDRA version 23.1 applied for Part A, MedDRA version 26.1 applied for Part B.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Part A: TEZ/IVA | Participants weighing <40 kg at Day 1 received TEZ 50 mg qd/IVA 75 mg q12h and the participants weighing >= 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. | 0 | 130 | 31 | 130 | 128 | 130 |
| EG001 | Part B: TEZ/IVA | 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. | 0 | 62 | 8 | 62 | 49 | 62 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Immune thrombocytopenia | Blood and lymphatic system disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Cystic fibrosis related diabetes | Congenital, familial and genetic disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Distal intestinal obstruction syndrome | Gastrointestinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Intestinal obstruction | Gastrointestinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Small intestinal obstruction | Gastrointestinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Bacterial disease carrier | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Chronic sinusitis | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Device related sepsis | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Pertussis | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Respiratory tract infection bacterial | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Upper respiratory tract infection bacterial | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Procedural haemorrhage | Injury, poisoning and procedural complications | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Radius fracture | Injury, poisoning and procedural complications | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Bacterial test positive | Investigations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Blood lactate dehydrogenase increased | Investigations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Pseudomonas test positive | Investigations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Pulmonary function test decreased | Investigations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Stenotrophomonas test positive | Investigations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Weight gain poor | Metabolism and nutrition disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Anxiety disorder | Psychiatric disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Hallucination | Psychiatric disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Personality change | Psychiatric disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Adenoidal hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Nasal polyps | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Sinus disorder | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Sinus polyp | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA 23.1,26.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Ear pain | Ear and labyrinth disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Bacterial disease carrier | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Pharyngitis streptococcal | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Bacterial test positive | Investigations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Forced expiratory volume decreased | Investigations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Pseudomonas test positive | Investigations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Staphylococcus test positive | Investigations | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Nasal polyps | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1,26.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 23.1,26.1 | Systematic Assessment |
|
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Monitor | Vertex Pharmaceuticals Incorporated | 617-341-6777 | medicalinfo@vrtx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Aug 19, 2020 | Oct 21, 2021 | SAP_001.pdf |
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000654124 | tezacaftor, ivacaftor drug combination |
| C545203 | ivacaftor |
Not provided
Not provided
Not provided
| Other |
|
|
| Male |
|
| Part B |
|
|
| Not Hispanic or Latino |
|
| Not collected per local regulations |
|
| Part B |
|
|
| Black or African American |
|
| Asian |
|
| Not collected per local regulations |
|
| Other |
|
| Part B |
|
|
| Participants |
|
|
|
|
|
|
|
|
|
|