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| ID | Type | Description | Link |
|---|---|---|---|
| 2018-000185-11 | EudraCT Number |
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The study evaluates the long-term safety and tolerability of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del mutation
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ELX/TEZ/IVA | Experimental | Treatment Period: Participants received ELX 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hrs (q12h) in the treatment period for 192 weeks. Extension Period: Participants from certain countries participated in extension period and continued to receive ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the extension period for 48 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ELX/TEZ/IVA | Drug | Fixed-dose combination (FDC) tablet for oral administration. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Treatment Period: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | From Day 1 up to Week 196 |
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 102/105 Efficacy Set | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. This analysis set included study 102 parent study participants who received Placebo-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. | From Baseline at Week 192 |
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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 |
|---|---|---|---|---|---|---|
| Banner University of Arizona Medical Center | Tucson | Arizona | 85724 | United States | ||
| University of Arkansas for Medical Sciences |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40209082 | Derived | Daines CL, Polineni D, Tullis E, Costa S, Linnemann RW, Mall MA, McKone EF, Quon BS, Ringshausen FC, Selvadurai H, Taylor-Cousar JL, Withers NJ, Sawicki GS, Lee T, Ahluwalia N, Morlando Geiger J, Jennings M, Tan YV, Waltz D, Ramsey B, Griese M; VX17-445-105 Study Group. Long-Term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Adults and Adolescents with Cystic Fibrosis and at Least One F508del Allele: A Phase 3 Open-Label Extension Study. Am J Respir Crit Care Med. 2025 Oct;211(10):1901-1914. doi: 10.1164/rccm.202411-2231OC. | |
| 37983082 |
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A total 507 participants were enrolled in this study from the parent studies VX17-445-102 (NCT03525444) and VX17-445-103 (NCT03525548). One participant was enrolled but did not receive any dose in this study.
The study was conducted as a single period i.e. treatment period in countries including United States (US) with commercially-available ELX/TEZ/IVA. The regional protocol for other countries without commercially-available ELX/TEZ/IVA was amended so that participants in these countries had the opportunity to participate for up to an additional 48 weeks in extension period.
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| ID | Title | Description |
|---|---|---|
| FG000 | ELX/TEZ/IVA | Treatment Period: Participants received ELX 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hrs (q12h) in the treatment period for 192 weeks. Extension Period: Participants from certain countries participated in extension period and continued to receive ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the extension period for 48 weeks. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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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 | Jul 29, 2020 | Jan 8, 2024 |
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| IVA | Drug | Tablet for oral administration. |
|
|
| Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 103/105 Efficacy Set | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. This analysis set included study 103 parent study participants who received TEZ/IVA-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. | From Baseline at Week 192 |
| Absolute Change in Sweat Chloride (SwCl) for 102/105 Efficacy Set | Sweat samples were collected using an approved collection device. This analysis set included study 102 parent study participants who received Placebo-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. | From Baseline at Week 192 |
| Absolute Change in Sweat Chloride (SwCl) for 103/105 Efficacy Set | Sweat samples were collected using an approved collection device. This analysis set included study 103 parent study participants who received TEZ/IVA-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. | From Baseline at Week 192 |
| Number of Pulmonary Exacerbations (PEx) for 102/105 Efficacy Set | Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms. | From Baseline at Week 192 |
| Number of Pulmonary Exacerbations (PEx) for 103/105 Efficacy Set | Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms. | From Baseline at Week 192 |
| Time to First PEx for 102/105 Efficacy Set | Time-to-first pulmonary exacerbation was analyzed using Kaplan-Meier estimates and expressed in terms of event-free probability. PEx was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms. | From Baseline at Week 192 |
| Time to First PEx for 103/105 Efficacy Set | Time-to-first pulmonary exacerbation was analyzed using Kaplan-Meier estimates and expressed in terms of event-free probability. PEx was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms. | From Baseline at Week 192 |
| Absolute Change in Body Mass Index (BMI) for 102/105 Efficacy Set | BMI was defined as weight in kilogram (kg) divided by height in square meter (m^2).This analysis set included study 102 parent study participants who received Placebo-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. | From Baseline at Week 192 |
| Absolute Change in Body Mass Index (BMI) for 103/105 Efficacy Set | BMI was defined as weight in kilogram (kg) divided by height in square meter (m^2).This analysis set included study 103 parent study participants who received TEZ/IVA-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. | From Baseline at Week 192 |
| Absolute Change in BMI Z-score for 102/105 Efficacy Set | BMI was defined as weight in kg divided by height in m^2. Z-score is a statistical measure to describe whether a mean was above or below the standard. BMI, adjusted for age and sex, was analyzed as BMI-for-age z-score. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Higher values are indicative of higher BMI. This analysis set included study 102 parent study participants who received Placebo-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. | From Baseline at Week 192 |
| Absolute Change in BMI Z-score for 103/105 Efficacy Set | BMI was defined as weight in kg divided by height in m^2. Z-score is a statistical measure to describe whether a mean was above or below the standard. BMI, adjusted for age and sex, was analyzed as BMI-for-age z-score. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Higher values are indicative of higher BMI. This analysis set included study 103 parent study participants who received TEZ/IVA-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. | From Baseline at Week 192 |
| Absolute Change in Body Weight for 102/105 Efficacy Set | This analysis set included study 102 parent study participants who received Placebo-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. | From Baseline at Week 192 |
| Absolute Change in Body Weight for 103/105 Efficacy Set | This analysis set included study 103 parent study participants who received TEZ/IVA and ELX/TEZ/IVA. | From Baseline at Week 192 |
| Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 102/105 Efficacy Set | 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. This analysis set included study 102 parent study participants who received Placebo-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. | From Baseline at Week 192 |
| Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 103/105 Efficacy Set | 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. This analysis set included study 103 parent study participants who received TEZ/IVA and ELX/TEZ/IVA. | From Baseline at Week 192 |
| Little Rock |
| Arkansas |
| 72205 |
| United States |
| Miller Children's Hospital / Long Beach Memorial | Long Beach | California | 90806 | United States |
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | United States |
| Valley Children's Hospital | Madera | California | 93636 | United States |
| Kaiser Permanente | Oakland | California | 94611 | United States |
| University of California Davis Medical Center | Sacramento | California | 95817 | United States |
| UCSF Gateway Medical Center | San Francisco | California | 94143 | United States |
| National Jewish Health | Denver | Colorado | 80206 | United States |
| University of Florida, Shands Hospital | Gainesville | Florida | 32610 | United States |
| Joe DiMaggio Cystic Fibrosis & Pulmonary Center | Hollywood | Florida | 33021 | United States |
| Nemours Children's Specialty Care | Jacksonville | Florida | 32207 | United States |
| Central Florida Pulmonary Group, PA | Orlando | Florida | 32803 | United States |
| Nemours Children's Hospital | Orlando | Florida | 32827 | United States |
| Tampa General Hospital Cardiac and Lung Transplant Clinic | Tampa | Florida | 33606 | United States |
| Children's Specialty Services at North Druid Hills | Atlanta | Georgia | 30324 | United States |
| Augusta University | Augusta | Georgia | 30912 | United States |
| Ann & Robert Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| Northwestern Memorial Hospital | Chicago | Illinois | 60611 | United States |
| Cystic Fibrosis Center, Children's Hospital of Illinois at OSF Saint Francis Medical Center | Peoria | Illinois | 61637 | United States |
| The University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| Tulane Medical Center | New Orleans | Louisiana | 70112 | United States |
| Maine Medical Partners | Portland | Maine | 04102 | United States |
| Massachusetts General Hospital Cystic Fibrosis Center Clinical Research Center | Boston | Massachusetts | 02114 | United States |
| Harper University Hospital | Detroit | Michigan | 48201 | United States |
| Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota | Minneapolis | Minnesota | 55404 | United States |
| Billings Clinic Hospital | Billings | Montana | 59101 | United States |
| Nebraska Medical Center | Omaha | Nebraska | 68198 | United States |
| Morristown Medical Center | Morristown | New Jersey | 07960 | United States |
| UNM Clinical and Translational Science Center | Albuquerque | New Mexico | 87131 | United States |
| The Cystic Fibrosis Center, Mount Sinai Beth Israel | New York | New York | 10003 | United States |
| New York Medical College | Valhalla | New York | 10595 | United States |
| UNC Marsico Clinical Research Center | Chapel Hill | North Carolina | 27517 | United States |
| Akron Children's Hospital | Akron | Ohio | 44308 | United States |
| UC Health Holmes | Cincinnati | Ohio | 45220 | United States |
| Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center | Cleveland | Ohio | 44106 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| Dayton Children's Hospital | Dayton | Ohio | 45404 | United States |
| ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center | Toledo | Ohio | 43606 | United States |
| University of Oklahoma Health Sciences Center | Oklahoma City | Oklahoma | 73104 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| Dell Children's Medical Center of Central Texas | Austin | Texas | 78723 | United States |
| The University of Texas Southwestern Medical Center | Dallas | Texas | 75390 | United States |
| Vermont Lung Center | Colchester | Vermont | 05446 | United States |
| University of Virginia Primary Care Center | Charlottesville | Virginia | 22908 | United States |
| Children's Hospital of the King's Daughters | Norfolk | Virginia | 23507 | United States |
| Children's Hospital of Richmond at VCU, Children's Pavilion | Richmond | Virginia | 23298 | United States |
| University of Washington Medical Center | Seattle | Washington | 98195 | United States |
| West Virginia University | Morgantown | West Virginia | 26506 | United States |
| University of Wisconsin Hospitals and Clinics | Madison | Wisconsin | 53792 | United States |
| CTSI Adult Translational Research Unit/Medical College of Wisconsin/Froedtert Hospital | Milwaukee | Wisconsin | 53226 | United States |
| Women & Children's Hospital | North Adelaide | Australia |
| The Royal Children's Hospital | Parkville | Australia |
| Mater Misericordiae Ltd | South Brisbane | Australia |
| The Children's Hospital at Westmead | Westmead | Australia |
| Westmead Hospital | Westmead | Australia |
| University of Graz | Graz | Austria |
| Medizinische Universität Innsbruck | Innsbruck | Austria |
| LKH - Universitätsklinikum der PMU Salzburg | Salzburg | Austria |
| Medizinische Universitat Wien | Vienna | Austria |
| Cliniques Universitaires de Bruxelles Hopital Erasme | Brussels | Belgium |
| Universitair Ziekenhuis Brussel - Campus Jette | Brussels | Belgium |
| UZ Antwerpen | Edegem | Belgium |
| Universitair Ziekenhuis Gent | Ghent | Belgium |
| Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg | Leuven | Belgium |
| University of Calgary Medical Clinic of the Foothills Medical Centre | Calgary | Canada |
| McGill University Health Centre, Glen Site, Montreal Children's Hospital | Montreal | Canada |
| Centre Hospitalier De L'Universite Laval | Québec | Canada |
| The Hospital for Sick Children | Toronto | Canada |
| British Columbia's Children's Hospital | Vancouver | Canada |
| St. Paul's Hospital | Vancouver | Canada |
| Fakultni Nemocnice Brno | Brno | Czechia |
| Fakultni nemocnice v Motole | Prague | Czechia |
| Centre Hospitalier Lyon Sud | Benite Cedex | France |
| Groupe Hospitaler Pellegrin, CHU De Bordeaux | Bordeaux | France |
| CHU Marseille - Hopital Nord | Marseille | France |
| CHU de Nice - Hopital Pasteur | Nice | France |
| Hopital Cochin | Paris | France |
| CHU de Rouen - Hopital Charles Nicolle | Rouen | France |
| Hopital Foch (Suresnes), Hopital Foch, Adultes | Suresnes | France |
| Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital | Erlangen | Germany |
| Justus-Leibig-Universitat Zentrum fur Kinderheilkunde und Jugendmedizin | Giessen | Germany |
| Hannover Medical School | Hanover | Germany |
| Johannes Gutenberg-Universitaet | Mainz | Germany |
| Dr. von Haunersches Kinderspital | München | Germany |
| Universitaetsklinikum Tuebingen Klinik fuer Kinder- und Jugendmedizin | Tübingen | Germany |
| University Hospital Wuerzburg | Würzburg | Germany |
| General Hospital of Attika "Sismanoglio"(Adult CF center, NHS) | Marousi | Greece |
| Azienda Ospedaliero Universitaria Ospendali Riuniti | Ancona | Italy |
| Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer | Florence | Italy |
| IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico | Genova | Italy |
| Azienda Ospedaliera Universitaria Policlinico G. Martino | Messina | Italy |
| Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico | Milan | Italy |
| Centro Regionale Fibrosi Cistica, A.O. Ospedale San Carlo | Potenza | Italy |
| Azienda Ospedaliera di Verona-Ospedale Civile Maggiore | Verona | Italy |
| Academic Medical Center | Amsterdam | Netherlands |
| University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis | Heidelberglaan | Netherlands |
| UMC St. Radboud | Nijmegen | Netherlands |
| Erasmus Medical Center | Rotterdam | Netherlands |
| HagaZiekenhuis van den Haag | The Hague | Netherlands |
| Karolinska Universitetssjukhuset, Huddinge | Stockholm | Sweden |
| Belfast City Hospital | Belfast | United Kingdom |
| Heart of England NHS Foundation Trust, Birmingham Heartlands Hospital | Birmingham | United Kingdom |
| Western General Hospital | Edinburgh | United Kingdom |
| Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital | Exeter | United Kingdom |
| Leeds General Infirmary | Leeds | United Kingdom |
| King's College Hospital | London | United Kingdom |
| Royal Manchester Children's Hospital | Manchester | United Kingdom |
| Southampton General Hospital | Southampton | United Kingdom |
| 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. |
| 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. |
| FG001 |
| Additional Participants for Cumulative TC Efficacy Set |
This reporting arm represents the three additional participants in Cumulative TC Efficacy Set 102/105 from parent study 102 which were not not enrolled in this study, but were included in the Cumulative TC efficacy 102/105 analysis set. |
| Open-Label Safety Set (OL-SS) |
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| OL-FAS 102/105 |
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| OL-FAS 103/105 |
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| Cumulative Triple Combination (TC) Efficacy Set 102/105 |
|
| Cumulative TC Efficacy Set 103/105 |
|
| COMPLETED |
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| NOT COMPLETED |
|
|
All participants who received at least one dose of the study drug in this open label study were included in the baseline characteristics.
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| ID | Title | Description |
|---|---|---|
| BG000 | ELX/TEZ/IVA | Treatment Period: Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 192 weeks. Extension Period: Participants from certain countries participated in extension period and continued to received ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the extension period for 48 weeks. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| ||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants | No |
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| Race/Ethnicity, Customized | Count of Participants | Participants | No |
| ||||||||||||||||||||||
| Race/Ethnicity, Customized | 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 | Treatment Period: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | The Open-Label Safety Set (OL-SS) included all participants who had received at least 1 dose of study drug in this open label study. | Posted | Count of Participants | Participants | No | From Day 1 up to Week 196 |
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| Secondary | Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 102/105 Efficacy Set | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. This analysis set included study 102 parent study participants who received Placebo-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. | OL-FAS is defined as all enrolled participants who received at least 1 dose of study drug in this open label study. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. Here "Number Analyzed" signifies participants who were evaluable for the specified category. Baseline was defined as the parent study baseline. | Posted | Least Squares Mean | Standard Error | percentage points | From Baseline at Week 192 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 103/105 Efficacy Set | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. This analysis set included study 103 parent study participants who received TEZ/IVA-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. | OL-FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. Here "Number Analyzed" signifies participants who were evaluable for the specified category. Baseline was defined as the parent study baseline. | Posted | Least Squares Mean | Standard Error | percentage points | From Baseline at Week 192 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in Sweat Chloride (SwCl) for 102/105 Efficacy Set | Sweat samples were collected using an approved collection device. This analysis set included study 102 parent study participants who received Placebo-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. | OL-FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. Here "Number Analyzed" signifies participants who were evaluable for the specified category. Baseline was defined as the parent study baseline. | Posted | Least Squares Mean | Standard Error | millimole per liter (mmol/L) | From Baseline at Week 192 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in Sweat Chloride (SwCl) for 103/105 Efficacy Set | Sweat samples were collected using an approved collection device. This analysis set included study 103 parent study participants who received TEZ/IVA-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. | OL-FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. Here "Number Analyzed" signifies participants who were evaluable for the specified category. Baseline was defined as the parent study baseline. | Posted | Least Squares Mean | Standard Error | mmol/L | From Baseline at Week 192 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Number of Pulmonary Exacerbations (PEx) for 102/105 Efficacy Set | Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms. | Cumulative TC Efficacy Set 102/105 included all participants who were randomized to TC ELX/TEZ/IVA and received at least 1 dose of study drug during the parent study 102 and/or received at least 1 dose of study drug during this open label study. Baseline was defined as the parent study baseline. | Posted | Number | PEx events | From Baseline at Week 192 |
|
| ||||||||||||||||||||||||||||||||||
| Secondary | Number of Pulmonary Exacerbations (PEx) for 103/105 Efficacy Set | Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms. | Cumulative TC Efficacy Set 103/105 included all participants who were randomized to TC ELX/TEZ/IVA and received at least 1 dose of study drug during the parent study 103 and/or received at least 1 dose of study drug during this open label study. Baseline was defined as the parent study baseline. | Posted | Number | PEx events | From Baseline at Week 192 |
|
| ||||||||||||||||||||||||||||||||||
| Secondary | Time to First PEx for 102/105 Efficacy Set | Time-to-first pulmonary exacerbation was analyzed using Kaplan-Meier estimates and expressed in terms of event-free probability. PEx was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms. | Cumulative TC Efficacy Set 102/105 included all participants who were randomized to TC ELX/TEZ/IVA and received at least 1 dose of study drug during the parent study 102 and/or received at least 1 dose of study drug during this open label study. Baseline was defined as the parent study baseline. | Posted | Median | 95% Confidence Interval | days | From Baseline at Week 192 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Time to First PEx for 103/105 Efficacy Set | Time-to-first pulmonary exacerbation was analyzed using Kaplan-Meier estimates and expressed in terms of event-free probability. PEx was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms. | Cumulative TC Efficacy Set 103/105 included all participants who were randomized to TC ELX/TEZ/IVA and received at least 1 dose of study drug during the parent study 103 and/or received at least 1 dose of study drug during this open label study. Baseline was defined as the parent study baseline. | Posted | Median | 95% Confidence Interval | days | From Baseline at Week 192 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in Body Mass Index (BMI) for 102/105 Efficacy Set | BMI was defined as weight in kilogram (kg) divided by height in square meter (m^2).This analysis set included study 102 parent study participants who received Placebo-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. | OL-FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. Here "Number Analyzed" signifies participants who were evaluable for the specified category. Baseline was defined as the parent study baseline. | Posted | Least Squares Mean | Standard Error | kilogram per meter square (kg/m^2) | From Baseline at Week 192 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in Body Mass Index (BMI) for 103/105 Efficacy Set | BMI was defined as weight in kilogram (kg) divided by height in square meter (m^2).This analysis set included study 103 parent study participants who received TEZ/IVA-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. | OL-FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. Here "Number Analyzed" signifies participants who were evaluable for the specified category. Baseline was defined as the parent study baseline. | Posted | Least Squares Mean | Standard Error | kg/m^2 | From Baseline at Week 192 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in BMI Z-score for 102/105 Efficacy Set | BMI was defined as weight in kg divided by height in m^2. Z-score is a statistical measure to describe whether a mean was above or below the standard. BMI, adjusted for age and sex, was analyzed as BMI-for-age z-score. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Higher values are indicative of higher BMI. This analysis set included study 102 parent study participants who received Placebo-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. | OL-FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were <=20 years of age at Baseline. Here "Number Analyzed" signifies participants who were evaluable for the specified category. Baseline was defined as the parent study baseline. | Posted | Least Squares Mean | Standard Error | z-score | From Baseline at Week 192 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in BMI Z-score for 103/105 Efficacy Set | BMI was defined as weight in kg divided by height in m^2. Z-score is a statistical measure to describe whether a mean was above or below the standard. BMI, adjusted for age and sex, was analyzed as BMI-for-age z-score. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Higher values are indicative of higher BMI. This analysis set included study 103 parent study participants who received TEZ/IVA-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. | OL-FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were <=20 years of age at Baseline. Here "Number Analyzed" signifies participants who were evaluable for the specified category. Baseline was defined as the parent study baseline. | Posted | Least Squares Mean | Standard Error | z-score | From Baseline at Week 192 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in Body Weight for 102/105 Efficacy Set | This analysis set included study 102 parent study participants who received Placebo-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. | OL-FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. Here "Number Analyzed" signifies participants who were evaluable for the specified category. Baseline was defined as the parent study baseline. | Posted | Least Squares Mean | Standard Error | kg | From Baseline at Week 192 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in Body Weight for 103/105 Efficacy Set | This analysis set included study 103 parent study participants who received TEZ/IVA and ELX/TEZ/IVA. | OL-FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. Here "Number Analyzed" signifies participants who were evaluable for the specified category. Baseline was defined as the parent study baseline. | Posted | Least Squares Mean | Standard Error | kg | From Baseline at Week 192 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 102/105 Efficacy Set | 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. This analysis set included study 102 parent study participants who received Placebo-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. | OL-FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. Here "Number Analyzed" signifies participants who were evaluable for the specified category. Baseline was defined as the parent study baseline. | Posted | Least Squares Mean | Standard Error | units on a scale | From Baseline at Week 192 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 103/105 Efficacy Set | 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. This analysis set included study 103 parent study participants who received TEZ/IVA and ELX/TEZ/IVA. | OL-FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. Here "Number Analyzed" signifies participants who were evaluable for the specified category. Baseline was defined as the parent study baseline. | Posted | Least Squares Mean | Standard Error | units on a scale | From Baseline at Week 192 |
|
|
Adverse events (AE) are reported separately for both Periods. Treatment period covers 1st dose till 196 weeks in treatment period; extension period covers 1st dose in extension period till safety follow-up or end of study, whichever occurs first. The actual total duration for AE collection is up to 222 weeks.
The study was conducted as a treatment period in countries including United States (US) with commercially-available ELX/TEZ/IVA. The regional protocol for other countries without commercially-available ELX/TEZ/IVA was amended so that participants in these countries had the opportunity to participate for up to an additional 48 weeks in extension period. The adverse events data has been reported for both the parts separately.
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 | Treatment Period: ELX/TEZ/IVA | Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 192 weeks. | 1 | 506 | 175 | 506 | 501 | 506 |
| EG001 | Extension Period: ELX/TEZ/IVA | Participants from certain countries participated in extension period and continued to receive ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the extension period for 48 weeks. | 0 | 11 | 0 | 11 | 7 | 11 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Leukocytosis | Blood and lymphatic system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Acute left ventricular failure | Cardiac disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Acute myocardial infarction | Cardiac disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Arrhythmia | Cardiac disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pericardial effusion | Cardiac disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pericarditis constrictive | Cardiac disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Postural orthostatic tachycardia syndrome | Cardiac disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Cystic fibrosis lung | Congenital, familial and genetic disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Thyroid mass | Endocrine disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Visual impairment | Eye disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Abdominal adhesions | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Abdominal hernia | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Cyclic vomiting syndrome | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Distal intestinal obstruction syndrome | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Duodenal ulcer haemorrhage | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Duodenitis | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Gastric fistula | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Gastric haemorrhage | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Ileus | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Intestinal obstruction | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pharyngo-oesophageal diverticulum | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Small intestinal obstruction | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Small intestinal perforation | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Drug withdrawal syndrome | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Physical deconditioning | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Cholecystitis acute | Hepatobiliary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Cholecystitis chronic | Hepatobiliary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Hypertransaminasaemia | Hepatobiliary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Drug hypersensitivity | Immune system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Bacteraemia | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Bacterial disease carrier | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Bronchopulmonary aspergillosis allergic | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Chest wall abscess | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Chronic sinusitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Infective exacerbation of bronchiectasis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Oesophageal candidiasis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Parainfluenzae virus infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Parotitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Pneumonia pseudomonal | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Pneumonia viral | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Post procedural infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Postoperative wound infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Pyelonephritis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Vascular device infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Alcohol poisoning | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Craniocerebral injury | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Fracture of penis | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Humerus fracture | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Lumbar vertebral fracture | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Pneumothorax traumatic | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Procedural pneumothorax | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Pulmonary contusion | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Radius fracture | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Rib fracture | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Road traffic accident | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Scapula fracture | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Skin injury | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Tibia fracture | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Toxicity to various agents | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Traumatic haemothorax | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Vascular access site pain | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Wound dehiscence | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Bacterial test positive | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Enterovirus test positive | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Human rhinovirus test positive | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Influenza A virus test positive | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Pulmonary function test decreased | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| SARS-CoV-2 test positive | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Diabetes mellitus inadequate control | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Diabetic ketoacidosis | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Hypercalcaemia | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Rhabdomyolysis | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Rheumatoid arthritis | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Adenocarcinoma of colon | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 25.1 | Systematic Assessment |
| |
| Philadelphia positive acute lymphocytic leukaemia | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 25.1 | Systematic Assessment |
| |
| Prostate cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 25.1 | Systematic Assessment |
| |
| Uterine leiomyoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 25.1 | Systematic Assessment |
| |
| Autonomic nervous system imbalance | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Cerebellar infarction | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Cerebral infarction | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Hepatic encephalopathy | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Miller Fisher syndrome | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Optic neuritis | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Abortion spontaneous | Pregnancy, puerperium and perinatal conditions | MedDRA 25.1 | Systematic Assessment |
| |
| Alcohol withdrawal syndrome | Psychiatric disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Anger | Psychiatric disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Anorexia nervosa | Psychiatric disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Bipolar disorder | Psychiatric disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Major depression | Psychiatric disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Mental disorder | Psychiatric disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Psychiatric decompensation | Psychiatric disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Psychotic disorder | Psychiatric disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Suicide attempt | Psychiatric disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Calculus urinary | Renal and urinary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Renal colic | Renal and urinary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Breast mass | Reproductive system and breast disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Testicular torsion | Reproductive system and breast disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Vaginal haemorrhage | Reproductive system and breast disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Haemothorax | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Lung infiltration | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pulmonary haemorrhage | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Rales | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Hypertensive urgency | Vascular disorders | MedDRA 25.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Palpitations | Cardiac disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Malaise | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Immunisation reaction | Immune system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Hordeolum | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Vulvovaginal mycotic infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Bacterial test positive | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Pulmonary function test decreased | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| SARS-CoV-2 test positive | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Lower respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Respiration abnormal | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Acne | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
|
Not provided
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 | Oct 27, 2022 | Jan 8, 2024 | SAP_001.pdf |
Not provided
| 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 |
|---|---|
| C000706587 | elexacaftor, ivacaftor, tezacaftor drug combination |
| C545203 | ivacaftor |
Not provided
Not provided
Not provided
| Not collected per local regulations |
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| American Indian or Alaska Native |
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| Other |
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| Not collected per local regulations |
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| Multiple |
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