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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-004134-29 | EudraCT Number |
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At Sponsor's Discretion
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This study will evaluate the long-term safety and tolerability of VX-659 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 |
|---|---|---|---|
| VX-659/TEZ/IVA TC | Experimental | Participants from parent studies VX17-659-102 (NCT03447249) or VX17-659-103 (NCT03460990) were administered VX-659 240 milligrams (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the TC treatment period for up to 96 weeks in the current study VX17-659-105. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VX-659/TEZ/IVA | Drug | Fixed-dose combination tablets for oral administration qd in the morning. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | From Day 1 up to 28 Days After Last Dose of Study Drug or to the Completion of Study Participation Date, Whichever Occurs First in the Current Study 659-105 (up to Week 100) |
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for Participants From the Parent Study 659-102 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. |
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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 | ||
| Stanford University |
| 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. | |
| 33331662 | Derived |
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This study was conducted in cystic fibrosis (CF) participants aged 12 years or older who participated in parent studies 659-102 or 659-103. Eligible participants from the parent studies were enrolled in the current study 659-105.
A total of 484 participants were enrolled from the parent studies VX17-659-102 (Study 659-102; NCT03447249) and VX17-659-103 (Study 659-103; NCT03460990). Out of which, 3 participants were enrolled but never dosed in the current study VX17-659-105 (Study 659-105). Therefore, the below results are presented for 481 participants.
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| ID | Title | Description |
|---|---|---|
| FG000 | VX-659/TEZ/IVA Triple Combination (TC) | Participants from the parent studies 659-102 or 659-103 were administered VX-659 240 milligrams (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the TC treatment period for up to 96 weeks in the current study 659-105. |
| 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 | Oct 23, 2018 | Sep 7, 2021 |
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| IVA | Drug | IVA tablet qd in the evening. |
|
|
| From Baseline at Week 72 (Study 659-105) |
| Absolute Change in ppFEV1 for Participants From the Parent Study 659-103 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. | From Baseline at Week 72 (Study 659-105) |
| Absolute Change in Sweat Chloride (SwCl) for Participants From the Parent Study 659-102 | Sweat samples were collected using an approved collection device. The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. | From Baseline at Week 24 (Study 659-105) |
| Absolute Change in SwCl for Participants From the Parent Study 659-103 | Sweat samples were collected using an approved collection device. The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. | From Baseline at Week 24 (Study 659-105) |
| Number of Pulmonary Exacerbations (PEx) for Participants From the Parent Study 659-102 | PEx was defined as treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for at least 4 sinopulmonary signs/symptoms. The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in the parent study 659-102 or/and VX-659/TEZ/IVA in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline except for Placebo - VX-659/TEZ/IVA category, for which the baseline was defined as study 659-105 baseline. | From Baseline up to Week 96 (Study 659-105) |
| Number of PEx for Participants From the Parent Study 659-103 | PEx was defined as treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for at least 4 sinopulmonary signs/symptoms. The analysis was planned to be reported for overall participants from the parent study 659-103, that is combined for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in the parent study 659-103 or/and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline except for TEZ/IVA - VX-659/TEZ/IVA category, for which the baseline was defined as study 659-105 baseline. | From Baseline up to Week 96 (Study 659-105) |
| Number of Participants With at Least One PEx for Participants From the Parent Study 659-102 | PEx was defined as treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for at least 4 sinopulmonary signs/symptoms. The time-to-first-PEx data were planned to be estimated using the Kaplan-Meier (KM) method. However, because way less than 50% of participants had events, median time-to-first event data were not estimable. Instead, the number of participants with at least one PEx event was assessed and reported separately for those in Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and the VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in the parent study 659-102 or/and VX-659/TEZ/IVA in the current study 659-105). Baseline was defined as the parent study baseline except for Placebo - VX-659/TEZ/IVA category, for which the baseline was defined as study 659-105 baseline. | From Baseline up to Week 96 (Study 659-105) |
| Number of Participants With at Least One PEx for Participants From the Parent Study 659-103 | PEx was defined as treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for at least 4 sinopulmonary signs/symptoms. The time-to-first-PEx data were planned to be estimated using the KM method. However, because way less than 50% of participants had events, median time-to-first-event data were not estimable. Instead, the number of participants with at least one PEx event was assessed and reported for all participants from the parent study 659-103, that is combined for those in the TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and the VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in the parent study 659-103 or/and in the current study 659-105). Baseline was defined as the parent study baseline except for TEZ/IVA - VX-659/TEZ/IVA category, for which the baseline was defined as study 659-105 baseline. | From Baseline up to Week 96 (Study 659-105) |
| Absolute Change in Body Mass Index (BMI) for Participants From the Parent Study 659-102 | BMI was defined as weight in kilograms (kg) divided by squared height in meters (m^2). The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. | From Baseline at Week 72 (Study 659-105) |
| Absolute Change in BMI for Participants From the Parent Study 659-103 | BMI was defined as weight in kg divided by squared height in meters (m^2). The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. | From Baseline at Week 72 (Study 659-105) |
| Absolute Change in BMI Z-score for Participants From the Parent Study 659-102 (Participants <=20 Years Old at Parent Study Baseline) | BMI was defined as weight in kg divided by squared height in meters (m^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. | From Baseline at Week 60 (Study 659-105) |
| Absolute Change in BMI Z-score for Participants From The Parent Study 659-103 (Participants <=20 Years Old at Parent Study Baseline) | BMI was defined as weight in kg divided by squared height in meters (m^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. | From Baseline at Week 60 (Study 659-105) |
| Absolute Change in Body Weight for Participants From the Parent Study 659-102 | The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. | From Baseline at Week 72 (Study 659-105) |
| Absolute Change in Body Weight for Participants From the Parent Study 659-103 | The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. | From Baseline at Week 72 (Study 659-105) |
| Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for Participants From the Parent Study 659-102 | 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. The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. | From Baseline at Week 72 (Study 659-105) |
| Absolute Change in CFQ-R Respiratory Domain Score for Participants From the Parent Study 659-103 | 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. The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. | From Baseline at Week 72 (Study 659-105) |
| Palo Alto |
| California |
| 94304 |
| United States |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | United States |
| Hartford Hospital | Hartford | Connecticut | 06102 | United States |
| Yale New Haven Medical Center | New Haven | Connecticut | 06511 | United States |
| University of Miami Miller School of Medicine | Miami | Florida | 33136 | United States |
| Nicklaus Children's Hospital | Miami | Florida | 33155 | United States |
| Orlando Health, Inc.- Arnold Palmer Hospital for Children (APH) | Orlando | Florida | 32806 | United States |
| Johns Hopkins All Children's Hospital Outpatient Care Center | St. Petersburg | Florida | 33701 | United States |
| St. Luke's CF Center of Idaho | Boise | Idaho | 83712 | United States |
| Cystic Fibrosis Center of Chicago | Glenview | Illinois | 60025 | United States |
| Advocate Children's Hospital - Park Ridge / North Suburban Pulmonary and Critical Care Consultants | Niles | Illinois | 60714 | United States |
| Indiana Clinical Research Center, IU Health University Hospital | Indianapolis | Indiana | 46202 | United States |
| The University of Iowa Hospitals and Clinics | Iowa City | Iowa | 52242 | United States |
| University of Kentucky | Lexington | Kentucky | 40536 | United States |
| Kosair Charities Pediatric Clinical Research Unit | Louisville | Kentucky | 40202 | United States |
| Johns Hopkins Hospital | Baltimore | Maryland | 21287 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| UMass Memorial Medical Center | Worcester | Massachusetts | 01655 | United States |
| Michigan Medicine | Ann Arbor | Michigan | 48109-5212 | United States |
| Spectrum Health Medical Group Adult Cystic Fibrosis Care Center | Grand Rapids | Michigan | 49546 | United States |
| University of Minnesota | Minneapolis | Minnesota | 55455 | United States |
| University of Mississippi Medical Center | Jackson | Mississippi | 39216 | United States |
| The Children's Mercy Hospital | Kansas City | Missouri | 64108 | United States |
| Washington University School of Medicine/ St. Louis Children's Hospital | St Louis | Missouri | 63110 | United States |
| Dartmouth Hitchcock, Manchester | Manchester | New Hampshire | 03104 | United States |
| Rutgers-Robert Wood Johnson Medical School | New Brunswick | New Jersey | 08901 | United States |
| Albany Medical College | Albany | New York | 12208 | United States |
| CF Therapeutics Development Center of Western New York | Buffalo | New York | 14203 | United States |
| Northwell Health, Long Island Jewish Medical Center | New Hyde Park | New York | 11040 | United States |
| Columbia University Medical Center | New York | New York | 10032 | United States |
| SUNY Upstate Medical University | Syracuse | New York | 13210 | United States |
| Clinical Research of Charlotte | Charlotte | North Carolina | 28277 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| Cincinnati Children's Hospital | Cincinnati | Ohio | 45229 | United States |
| Santiago Reyes, M.D. | Oklahoma City | Oklahoma | 73112 | United States |
| Oregon Health & Science University | Portland | Oregon | 97239 | United States |
| Drexel University College of Medicine / Drexel Adult Cystic Fibrosis Center | Philadelphia | Pennsylvania | 19107 | United States |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15224 | United States |
| Sanford Children's Specialty Clinic | Sioux Falls | South Dakota | 57105 | United States |
| University of Tennessee Medical Center- Adult Cystic Fibrosis Clinic | Knoxville | Tennessee | 37920 | United States |
| Children's Foundation Research Center / Le Bonheur Children's Hospital | Memphis | Tennessee | 38103 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | United States |
| Cook Children's Medical Center | Fort Worth | Texas | 76104 | United States |
| Texas Children's Hospital | Houston | Texas | 77030 | United States |
| University of Utah/ Primary Children's Medical Center | Salt Lake City | Utah | 84132 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| Providence Pediatric Pulmonary & Cystic Fibrosis Clinic | Spokane | Washington | 99204 | United States |
| Royal Adelaide Hospital | Adelaide | Australia |
| The Prince Charles Hospital | Chermside | Australia |
| Alfred Hospital | Melbourne | Australia |
| Institute for Respiratory Health, Sir Charles Gairdner Hospital | Nedlands | Australia |
| John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital | New Lambton | Australia |
| Telethon Kids Institute | Perth | Australia |
| Sydney Children's Hospital | Randwick | Australia |
| Lady Cilento Children's Hospital | South Brisbane | Australia |
| Stollery Children's Hospital | Edmonton | Canada |
| Queen Elizabeth II Health Sciences Center | Halifax | Canada |
| St. Michael's Hospital | Toronto | Canada |
| Juliane Marie Center, Rigshospitalet | Copenhagen | Denmark |
| Charite Paediatric Pulmonology Department | Berlin | Germany |
| Universitaetsklinkum Koeln, CF-Studienzentrum | Cologne | Germany |
| Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen | Essen | Germany |
| Clinic of J.W. Goethe University | Frankfurt | Germany |
| Medizinische Hochschule Hannover | Hanover | Germany |
| Mukeviszidose-Zentrum am Universitatsklinikum Jena, Klinik fuer Kinder- und Jugendmedizin | Jena | Germany |
| Universitatsklinikum Schleswig-Holstein, Klinik für Kinder- und Jugendmedizin | Lübeck | Germany |
| Klinikum Innenstadt, University of Munich | München | Germany |
| Pneumologische Praxis Pasing | München | Germany |
| Cork University Hospital | Cork | Ireland |
| Beaumont Hospital | Dublin | Ireland |
| Our Lady's Children's Hospital | Dublin | Ireland |
| St. Vincent's University Hospital | Dublin | Ireland |
| Temple Street Children's University Hospital | Dublin | Ireland |
| University Hospital Galway | Galway | Ireland |
| University Hospital Limerick | Limerick | Ireland |
| Lady Davis Carmel Medical Center | Haifa | Israel |
| Rambam Health Care Campus, Liver Unit | Haifa | Israel |
| Pediatrics Hadassah Medical Center | Jerusalem | Israel |
| Schneider Children's Medical Center | Petah Tikva | Israel |
| Sheba Medical Center | Tel Litwinsky | Israel |
| Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY | Łomianki | Poland |
| Hospital Universitari Vall d Hebron | Barcelona | Spain |
| Hospital Universitari Vall d´Hebron Servicio de Broncoscopia | Barcelona | Spain |
| Hospital Universitario 12 de Octubre | Madrid | Spain |
| Hospital Universitario Infantil La Paz | Madrid | Spain |
| Parc Tauli Sabadell Hospital Universitari | Sabadell | Spain |
| Hospital Universitario Virgen del Rocio | Seville | Spain |
| Hospital Universitario y Politecnico La Fe | Valencia | Spain |
| Lindenhofspital - Quartier Bleu | Bern | Switzerland |
| Kinderspital Zuerich | Zurich | Switzerland |
| Papworth Hospital NHS Foundation Trust, Papworth Everard | Cambridge | United Kingdom |
| Clinical Research Facility, Queen Elizabeth University Hospital | Glasgow | United Kingdom |
| St. James University Hospital | Leeds | United Kingdom |
| Liverpool Head and Chest Hospital | Liverpool | United Kingdom |
| Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital | London | United Kingdom |
| Wythenshaw e Hospital | Manchester | United Kingdom |
| The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary | Newcastle upon Tyne | United Kingdom |
| Wolfson Cystic Fibrosis Unit, City Campus | Nottingham | United Kingdom |
| All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough | Penarth | United Kingdom |
| 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. |
| COMPLETED |
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| NOT COMPLETED |
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Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | VX-659/TEZ/IVA TC | Participants from parent studies 659-102 or 659-103 were administered VX-659 240 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the TC treatment period for up to 96 weeks in the current study 659-105. |
| 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 |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| 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 | Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Open label safety set (OL-SS) included all participants who received at least 1 dose of study drug in the current study 659-105. | Posted | Number | participants | From Day 1 up to 28 Days After Last Dose of Study Drug or to the Completion of Study Participation Date, Whichever Occurs First in the Current Study 659-105 (up to Week 100) |
|
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| ||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for Participants From the Parent Study 659-102 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. | Open label full analysis set (OL-FAS) included all rolled over participants from the parent study 659-102 who received at least 1 dose of study drug in the current study 659-105. Here, "number analyzed" signifies participants who were evaluable for the specified category. | Posted | Mean | Standard Deviation | percentage points | From Baseline at Week 72 (Study 659-105) |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in ppFEV1 for Participants From the Parent Study 659-103 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. | OL-FAS included all rolled over participants from the parent study 659-103 who received at least 1 dose of study drug in the current study 659-105. Here, "number analyzed" signifies participants who were evaluable for the specified category. | Posted | Mean | Standard Deviation | percentage points | From Baseline at Week 72 (Study 659-105) |
|
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| Secondary | Absolute Change in Sweat Chloride (SwCl) for Participants From the Parent Study 659-102 | Sweat samples were collected using an approved collection device. The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. | OL-FAS included all rolled over participants from the parent study 659-102 who received at least 1 dose of study drug in the current study 659-105. Here, "number analyzed" signifies participants who were evaluable for the specified category. | Posted | Mean | Standard Deviation | millimole per liter (mmol/L) | From Baseline at Week 24 (Study 659-105) |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in SwCl for Participants From the Parent Study 659-103 | Sweat samples were collected using an approved collection device. The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. | OL-FAS included all rolled over participants from the parent study 659-103 who received at least 1 dose of study drug in the current study 659-105. Here, "number analyzed" signifies participants who were evaluable for the specified category. | Posted | Mean | Standard Deviation | mmol/L | From Baseline at Week 24 (Study 659-105) |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Number of Pulmonary Exacerbations (PEx) for Participants From the Parent Study 659-102 | PEx was defined as treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for at least 4 sinopulmonary signs/symptoms. The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in the parent study 659-102 or/and VX-659/TEZ/IVA in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline except for Placebo - VX-659/TEZ/IVA category, for which the baseline was defined as study 659-105 baseline. | The cumulative TC efficacy set for PEx analysis included all participants who were randomized to VX-659/TEZ/IVA arm and received at least one dose of study drug during the parent study 659-102 and/or received at least one dose of study drug during the current study 659-105. Here, "number analyzed" signifies participants who were evaluable for the specified category. | Posted | Number | PEx events | From Baseline up to Week 96 (Study 659-105) |
| |||||||||||||||||||||||||||||||||||
| Secondary | Number of PEx for Participants From the Parent Study 659-103 | PEx was defined as treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for at least 4 sinopulmonary signs/symptoms. The analysis was planned to be reported for overall participants from the parent study 659-103, that is combined for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in the parent study 659-103 or/and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline except for TEZ/IVA - VX-659/TEZ/IVA category, for which the baseline was defined as study 659-105 baseline. | The cumulative TC efficacy set for PEx analysis included all participants who were randomized to VX-659/TEZ/IVA arm and received at least one dose of study drug during the parent study 659-103 and/or received at least one dose of study drug during the current study 659-105. | Posted | Number | PEx events | From Baseline up to Week 96 (Study 659-105) |
|
| ||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With at Least One PEx for Participants From the Parent Study 659-102 | PEx was defined as treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for at least 4 sinopulmonary signs/symptoms. The time-to-first-PEx data were planned to be estimated using the Kaplan-Meier (KM) method. However, because way less than 50% of participants had events, median time-to-first event data were not estimable. Instead, the number of participants with at least one PEx event was assessed and reported separately for those in Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and the VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in the parent study 659-102 or/and VX-659/TEZ/IVA in the current study 659-105). Baseline was defined as the parent study baseline except for Placebo - VX-659/TEZ/IVA category, for which the baseline was defined as study 659-105 baseline. | The cumulative TC efficacy set for PEx analysis included all participants who were randomized to VX-659/TEZ/IVA arm and received at least one dose of study drug during the parent study 659-102 and/or received at least one dose of study drug during the current study 659-105. Here, "number analyzed" signifies participants who were evaluable for the specified category. | Posted | Number | participants | From Baseline up to Week 96 (Study 659-105) |
| |||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With at Least One PEx for Participants From the Parent Study 659-103 | PEx was defined as treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for at least 4 sinopulmonary signs/symptoms. The time-to-first-PEx data were planned to be estimated using the KM method. However, because way less than 50% of participants had events, median time-to-first-event data were not estimable. Instead, the number of participants with at least one PEx event was assessed and reported for all participants from the parent study 659-103, that is combined for those in the TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and the VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in the parent study 659-103 or/and in the current study 659-105). Baseline was defined as the parent study baseline except for TEZ/IVA - VX-659/TEZ/IVA category, for which the baseline was defined as study 659-105 baseline. | The cumulative TC efficacy set for PEx analysis included all participants who were randomized to VX-659/TEZ/IVA arm and received at least one dose of study drug during the parent study 659-103 and/or received at least one dose of study drug during the current study 659-105. | Posted | Number | participants | From Baseline up to Week 96 (Study 659-105) |
| |||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in Body Mass Index (BMI) for Participants From the Parent Study 659-102 | BMI was defined as weight in kilograms (kg) divided by squared height in meters (m^2). The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. | OL-FAS included all rolled over participants from the parent study 659-102 who received at least 1 dose of study drug in the current study 659-105. Here, "number analyzed" signifies participants who were evaluable for the specified category. | Posted | Mean | Standard Deviation | kilogram per meter square (kg/m^2) | From Baseline at Week 72 (Study 659-105) |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in BMI for Participants From the Parent Study 659-103 | BMI was defined as weight in kg divided by squared height in meters (m^2). The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. | OL-FAS included all rolled over participants from the parent study 659-103 who received at least 1 dose of study drug in the current study 659-105. Here, "number analyzed" signifies participants who were evaluable for the specified category. | Posted | Mean | Standard Deviation | kg/m^2 | From Baseline at Week 72 (Study 659-105) |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in BMI Z-score for Participants From the Parent Study 659-102 (Participants <=20 Years Old at Parent Study Baseline) | BMI was defined as weight in kg divided by squared height in meters (m^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. | OL-FAS included all rolled over participants from the parent study 659-102 who were <=20 years old at parent study baseline and received at least 1 dose of study drug in the current study 659-105. Here, "number analyzed" signifies participants who were evaluable for the specified category. | Posted | Mean | Standard Deviation | z-score | From Baseline at Week 60 (Study 659-105) |
| ||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in BMI Z-score for Participants From The Parent Study 659-103 (Participants <=20 Years Old at Parent Study Baseline) | BMI was defined as weight in kg divided by squared height in meters (m^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. | OL-FAS included all rolled over participants from the parent study 659-103 who were <=20 years old at parent study baseline and received at least 1 dose of study drug in the current study 659-105. Here, "number analyzed" signifies participants who were evaluable for the specified category. | Posted | Mean | Standard Deviation | z-score | From Baseline at Week 60 (Study 659-105) |
| ||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in Body Weight for Participants From the Parent Study 659-102 | The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. | OL-FAS included all rolled over participants from the parent study 659-102 who received at least 1 dose of study drug in the current study 659-105. Here, "number analyzed" signifies participants who were evaluable for the specified category. | Posted | Mean | Standard Deviation | kg | From Baseline at Week 72 (Study 659-105) |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in Body Weight for Participants From the Parent Study 659-103 | The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. | OL-FAS included all rolled over participants from the parent study 659-103 who received at least 1 dose of study drug in the current study 659-105. Here, "number analyzed" signifies participants who were evaluable for the specified category. | Posted | Mean | Standard Deviation | kg | From Baseline at Week 72 (Study 659-105) |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for Participants From the Parent Study 659-102 | 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. The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. | OL-FAS included all rolled over participants from the parent study 659-102 who received at least 1 dose of study drug in the current study 659-105. Here, "number analyzed" signifies participants who were evaluable for the specified category. | Posted | Mean | Standard Deviation | units on a scale | From Baseline at Week 72 (Study 659-105) |
| ||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in CFQ-R Respiratory Domain Score for Participants From the Parent Study 659-103 | 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. The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. | OL-FAS included all rolled over participants from the parent study 659-103 who received at least 1 dose of study drug in the current study 659-105. Here, "number analyzed" signifies participants who were evaluable for the specified category. | Posted | Mean | Standard Deviation | units on a scale | From Baseline at Week 72 (Study 659-105) |
|
|
From Day 1 up to 28 days After Last Dose of Study Drug or to the Completion of Study Participation Date, Whichever Occurs First in the Current Study 659-105 (up to Week 100)
Not provided
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 | VX-659/TEZ/IVA TC | Participants from parent studies 659-102 or 659-103 were administered VX-659 240 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the TC treatment period for up to 96 weeks in the current study 659-105. | 2 | 481 | 99 | 481 | 446 | 481 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cardiac failure acute | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Restrictive cardiomyopathy | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cystic fibrosis related diabetes | Congenital, familial and genetic disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Distal intestinal obstruction syndrome | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Duodenitis | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Intestinal obstruction | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Mechanical ileus | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Generalised oedema | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Systemic inflammatory response syndrome | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Autoimmune hepatitis | Hepatobiliary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Bile duct stone | Hepatobiliary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Biliary colic | Hepatobiliary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cholangitis | Hepatobiliary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cholecystitis | Hepatobiliary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cholecystitis chronic | Hepatobiliary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Anaphylactic reaction | Immune system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Bacterial disease carrier | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Bronchitis bacterial | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| H1N1 influenza | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Lower respiratory tract infection bacterial | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Mastoiditis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Medical device site cellulitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Pneumonia pseudomonal | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Vascular device infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Alcohol poisoning | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Craniocerebral injury | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Forearm fracture | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Intentional overdose | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Stoma site extravasation | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Toxicity to various agents | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Pulmonary function test decreased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Diabetes mellitus | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Diabetes mellitus inadequate control | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Compartment syndrome | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Device leakage | Product Issues | MedDRA 23.0 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Breathing-related sleep disorder | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Delirium | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Suicidal ideation | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hydronephrosis | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pelvi-ureteric obstruction | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Granulomatous pneumonitis | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Increased bronchial secretion | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pleuritic pain | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Respiration abnormal | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 23.0 | 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 | Feb 4, 2020 | Sep 7, 2021 | 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 |
|---|---|
| C545203 | ivacaftor |
Not provided
Not provided
Not provided
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
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