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| ID | Type | Description | Link |
|---|---|---|---|
| 2018-004652-38 | EudraCT Number |
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This study evaluated the long-term safety and tolerability of elexacaftor (ELX), tezacaftor (TEZ), and ivacaftor (IVA) triple combination (TC) treatment in participants with cystic fibrosis (CF).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ELX/TEZ/IVA | Experimental | Part A: Participants received ELX 200 milligram (mg) once daily (qd),TEZ 100 mg qd, and IVA 150 mg every 12 hours (q12h) in the treatment period for 96 weeks. Part B: Participants from certain countries participated in Part B and continued to received ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment 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 |
|---|---|---|
| Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | From Baseline through Week 100 |
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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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Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent research/clinical-trial-data-sharing
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A total 458 participants were enrolled from the parent study VX17-659-105 (NCT03447262). One participant was enrolled but did not receive any dose in this study.
The study was conducted as a single part study (Part A) in countries including United States (US) with commercially-available ELX/TEZ/IVA. The regional protocol for 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 Part B.
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| ID | Title | Description |
|---|---|---|
| FG000 | ELX/TEZ/IVA | Part A: Participants received ELX 200 milligram (mg) once daily (qd),TEZ 100 mg qd, and IVA 150 mg every 12 hours (q12h) in the treatment period for 96 weeks. Part B: Participants from certain countries participated in Part B and continued to received ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment 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 | Mar 8, 2021 | Jun 6, 2023 |
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| IVA | Drug | Tablet for oral administration. |
|
|
| 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 Hospital | New Haven | Connecticut | 06510 | United States |
| University of Miami Miller School of Medicine | Miami | Florida | 33136 | United States |
| Nicklaus Children's Hospital | Miami | Florida | 33155 | United States |
| Arnold Palmer Hospital Pulmonary and Sleep Medicine | 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 | 83702 | United States |
| Cystic Fibrosis Institute | Glenview | Illinois | 60025 | United States |
| Advocate Children's Hospital - Park Ridge / North Suburban Pulmonary and Critical Care Consultants | Niles | Illinois | 60714 | United States |
| Indiana University | 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 |
| 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 Medical Center | Lebanon | New Hampshire | 03756 | United States |
| Rutgers Robert Wood Johnson Medical School | New Brunswick | New Jersey | 08901 | United States |
| Albany Medical College, Pulmonary and Critical Care Medicine | Albany | New York | 12208 | United States |
| Lung and Cystic Fibrosis Center at Women and Children's Hospital of Buffalo | Buffalo | New York | 14222 | 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 Medical Center | Cincinnati | Ohio | 45229 | United States |
| Santiago Reyes, M.D. | Oklahoma City | Oklahoma | 73112 | United States |
| Oregon Health & Science University | Portland | Oregon | 97239 | United States |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15224 | United States |
| Sanford Children's Speciality Clinic | Sioux Falls | South Dakota | 57105 | United States |
| University of Tennessee Medical Center | 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, VIC | Australia |
| Institute for Respiratory Health | Nedlands | Australia |
| Perth Children's Hospital | Nedlands | Australia |
| John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital | New Lambton | Australia |
| Queensland 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 |
| Johann Wolfgang Goethe University | Frankfurt | Germany |
| Hannover Medical School | Hanover | Germany |
| Universitaetsklinikum Jena, Mukoviszidose-Zentrum | Jena | Germany |
| Universitatsklinikum Schleswig-Holstein, Klinik für Kinder- und Jugendmedizin | Lübeck | Germany |
| Klinikum Innenstadt, University of Munich | München | Germany |
| Pneumologisches Studienzentrum Muenchen-West | München | Germany |
| Cork University Hospital | Cork | Ireland |
| Beaumont Hospital | Dublin | Ireland |
| Children's Health Ireland at Crumlin | Dublin | Ireland |
| Children's Health Ireland at Temple Street | Dublin | Ireland |
| St. Vincent's University Hospital | Dublin | Ireland |
| University Hospital Galway | Galway | Ireland |
| University Hospital Limerick (Adults) | Limerick | Ireland |
| Lady Davis Carmel Medical Center | Haifa | Israel |
| Pediatric Pulmonary Unit Rambam Medical Center | Haifa | Israel |
| Hadassah Medical Organization | 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 |
| NHS Greater Glasgow and Clyde, Gartnavel General Hospital | Glasgow | United Kingdom |
| St. James University Hospital | Leeds | United Kingdom |
| Liverpool Heart and Chest Hospital | Liverpool | United Kingdom |
| Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital | London | United Kingdom |
| Wythenshawe Hospital | Manchester | United Kingdom |
| The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary | Newcastle upon Tyne | United Kingdom |
| Nottingham University Hospitals NHS Trust, Queens Medical Center | 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. |
| Safety Set |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
All participants who received at least one dose of the study drug were included in the baseline characteristics.
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| ID | Title | Description |
|---|---|---|
| BG000 | ELX/TEZ/IVA | Part A: Participants received ELX 200 milligram (mg) once daily (qd),TEZ 100 mg qd, and IVA 150 mg every 12 hours (q12h) in the treatment period for 96 weeks. Part B: Participants from certain countries participated in Part B and continued to received ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment 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 |
| |||||||||||||||||||||||
| Race/Ethnicity, Customized | Number | participants |
| |||||||||||||||||||||||
| Race/Ethnicity, Customized | 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 | Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Safety set for included all participants who received at least 1 dose of study drug. | Posted | Number | participants | From Baseline through Week 100 |
|
|
|
Day 1 Through Safety Follow-up Visit (up to Week 100 for Part A, up to Week 52 for Part B)
The study was conducted as a single part study (Part A) in countries including United States (US) with commercially-available ELX/TEZ/IVA. The regional protocol for 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 Part B. The adverse events data has been reported for both the parts separately. MedDRA 25.0 applied for Part A and MedDRA 25.1 applied for Part B.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Part A: ELX/TEZ/IVA | Participants received ELX 200 mg qd, TEZ 100 mg qd, and IVA 150 mg q12h in the treatment period for 96 weeks. | 0 | 457 | 75 | 457 | 404 | 457 |
| EG001 | Part B: ELX/TEZ/IVA | Participants from certain countries participated in Part B and continued to received ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks. | 0 | 66 | 4 | 66 | 45 | 66 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cystic fibrosis related diabetes | Congenital, familial and genetic disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Distal intestinal obstruction syndrome | Gastrointestinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Duodenitis | Gastrointestinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Intestinal obstruction | Gastrointestinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Oesophageal obstruction | Gastrointestinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Pancreatitis acute | Gastrointestinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Proctitis | Gastrointestinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Small intestinal obstruction | Gastrointestinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Umbilical hernia | Gastrointestinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Hypothermia | General disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Vascular device occlusion | General disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Abdominal abscess | Infections and infestations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Campylobacter gastroenteritis | Infections and infestations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Catheter site infection | Infections and infestations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Herpes simplex pharyngitis | Infections and infestations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Respiratory tract infection bacterial | Infections and infestations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Respiratory tract infection viral | Infections and infestations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Ligament rupture | Injury, poisoning and procedural complications | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Post procedural fistula | Injury, poisoning and procedural complications | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Stoma site discharge | Injury, poisoning and procedural complications | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Human rhinovirus test positive | Investigations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Influenza A virus test positive | Investigations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| SARS-CoV-2 test positive | Investigations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Abnormal loss of weight | Metabolism and nutrition disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Metabolic disorder | Metabolism and nutrition disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Type 1 diabetes mellitus | Metabolism and nutrition disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Joint instability | Musculoskeletal and connective tissue disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Adenocarcinoma of colon | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Intraductal proliferative breast lesion | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Papillary thyroid cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Piriformis syndrome | Nervous system disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Confusional state | Psychiatric disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Conversion disorder | Psychiatric disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Generalised anxiety disorder | Psychiatric disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Substance-induced mood disorder | Psychiatric disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Suicidal ideation | Psychiatric disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Hydronephrosis | Renal and urinary disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Ureterolithiasis | Renal and urinary disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Ovarian cyst ruptured | Reproductive system and breast disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Nasal polyps | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Immunisation reaction | Immune system disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Respiration abnormal | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0, 25.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 25.0, 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 | Jul 12, 2021 | Jun 6, 2023 | SAP_001.pdf |
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000706587 | elexacaftor, ivacaftor, tezacaftor drug combination |
| C545203 | ivacaftor |
Not provided
Not provided
Not provided
| Not collected per local regulations |
|
| Asian |
|
| American Indian or Alaska Native |
|
| Native Hawaiian or Other Pacific Islander |
|
| Not collected per local regulations |
|
| Multiple |
|