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| ID | Type | Description | Link |
|---|---|---|---|
| 2021-000694-85 | EudraCT Number |
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The purpose of this study is to evaluate the efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who are homozygous for F508del, heterozygous for F508del and a gating (F/G) or residual function (F/RF) mutation, or have at least 1 other TCR CF transmembrane conductance regulator (CFTR) gene mutation and no F508del mutation.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ELX/TEZ/IVA | Active Comparator | Following elexacftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) run-in period of 4 weeks, participants received ELX 200 milligram (mg) once daily (qd) /TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 52 weeks. |
|
| VX-121/TEZ/D-IVA | Experimental | Following ELX/TEZ/IVA run-in period of 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VX-121/TEZ/D-IVA | Drug | Fixed-dose combination tablets for oral administration. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change in Percent Predicted Forced Expiratory Volume in 1second (ppFEV1) | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | From Baseline Through Week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change in Sweat Chloride (SwCl) | Sweat samples were collected using an approved collection device. | From Baseline Through Week 24 |
| Percentage of Participants With SwCl <60 Millimole Per Liter (mmol/L) (Pooled With Data From Study VX20-121-102) |
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Key Inclusion Criteria:
Participant has one of the following genotypes:
Forced expiratory volume in 1 second (FEV1) value >=40% and <=90% of predicted mean for age, sex, and height for participants currently receiving CFTR protein modulator therapy; FEV1 >=40% and <=80% for participants not currently receiving CFTR protein modulator therapy
Key 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 | ||
| Banner University of Arizona Medical Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39756424 | Derived | Keating C, Yonker LM, Vermeulen F, Prais D, Linnemann RW, Trimble A, Kotsimbos T, Mermis J, Braun AT, O'Carroll M, Sutharsan S, Ramsey B, Mall MA, Taylor-Cousar JL, McKone EF, Tullis E, Floreth T, Michelson P, Sosnay PR, Nair N, Zahigian R, Martin H, Ahluwalia N, Lam A, Horsley A; VX20-121-102 Study Group; VX20-121-103 Study Group. Vanzacaftor-tezacaftor-deutivacaftor versus elexacaftor-tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years and older (SKYLINE Trials VX20-121-102 and VX20-121-103): results from two randomised, active-controlled, phase 3 trials. Lancet Respir Med. 2025 Mar;13(3):256-271. doi: 10.1016/S2213-2600(24)00411-9. Epub 2025 Jan 2. | |
| 37983082 |
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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 of 597 participants were enrolled in this study, of which 24 were included in the run-in period but were not dosed in treatment period. Therefore, results are presented for only 573 participants dosed in the treatment period.
This study was conducted in cystic fibrosis (CF) participants aged 12 years or older. It was pre-specified in the protocol to combine the data from this study with study VX20-121-102 (NCT05033080) for selected outcome measures.
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| ID | Title | Description |
|---|---|---|
| FG000 | ELX/TEZ/IVA | Following elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) run-in period of 4 weeks, participants received ELX 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 52 weeks. |
| FG001 | VX-121/TEZ/D-IVA |
| 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 | Aug 19, 2021 | May 10, 2024 |
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| ELX/TEZ/IVA | Drug | Fixed-dose combination tablets for oral administration. |
|
|
| IVA | Drug | Tablet for oral administration. |
|
|
| Placebo (matched to VX-121/TEZ/D-IVA) | Drug | Placebo matched to VX-121/TEZ/D-IVA for oral administration. |
|
| Placebo (matched to ELX/TEZ/IVA) | Drug | Placebo matched to ELX/TEZ/IVA for oral administration. |
|
| Placebo (matched to IVA) | Drug | Placebo matched to IVA for oral administration. |
|
Sweat samples were collected using an approved collection device. |
| From Baseline Through Week 24 |
| Percentage of Participants With SwCl <30 mmol/L (Pooled With Data From Study VX20-121-102) | Sweat samples were collected using an approved collection device. | From Baseline Through Week 24 |
| Tucson |
| Arizona |
| 85724 |
| United States |
| University of Arkansas for Medical Sciences | Little Rock | Arkansas | 72205 | United States |
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | United States |
| Children's Hospital of Orange County | Orange | California | 92868 | United States |
| University of California Davis Medical Center | Sacramento | California | 95817 | United States |
| Children's Hospital of Colorado | Aurora | Colorado | 80045 | United States |
| National Jewish Health | Denver | Colorado | 80206 | United States |
| Yale New Haven Hospital | New Haven | Connecticut | 06510 | United States |
| Nemours Children's Specialty Care | Jacksonville | Florida | 32207 | United States |
| University of Miami Miller School of Medicine | Miami | Florida | 33136 | United States |
| Central Florida Pulmonary Group, PA | Orlando | Florida | 32803 | United States |
| Nemours Children's Specialty Care, Pensacola | Pensacola | Florida | 32514 | United States |
| Johns Hopkins All Children's Hospital Outpatient Care Center | St. Petersburg | Florida | 33701 | United States |
| The Emory Clinic at Chantilly | Atlanta | Georgia | 30324 | United States |
| St. Luke's Cystic Fibrosis Center of Idaho | Boise | Idaho | 83702 | United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| Northwestern Memorial Hospital | Chicago | Illinois | 60611 | United States |
| Indiana University | Indianapolis | Indiana | 46202 | United States |
| Riley Hospital for Children at Indiana University Health | Indianapolis | Indiana | 46202 | United States |
| University of Kentucky | Lexington | Kentucky | 40536 | 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 Rsearch Center | Boston | Massachusetts | 02114 | 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 |
| Harper University Hospital | Detroit | Michigan | 48201 | United States |
| Spectrum Health Medical Group Adult Cystic Fibrosis Care Center | Grand Rapids | Michigan | 49546 | 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 | Missouri | 63110 | United States |
| Billings Clinic | Billings | Montana | 59101 | United States |
| Morristown Medical Center | Morristown | New Jersey | 07960 | 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 |
| Wake Forest Baptist Health | Winston-Salem | North Carolina | 27104 | United States |
| Akron Children's Hospital | Akron | Ohio | 44308 | 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 |
| Santiago Reyes, M.D. | Oklahoma City | Oklahoma | 73112 | United States |
| Oregon Health & Science University | Portland | Oregon | 97239 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | United States |
| Prisma Health Richland Campus | Columbia | South Carolina | 29203 | United States |
| Sanford Children's Speciality Clinic | Sioux Falls | South Dakota | 57105 | United States |
| University of Tennessee Medical Center | Knoxville | Tennessee | 37920 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | United States |
| The University of Texas Southwestern Medical Center | Dallas | Texas | 75390-8558 | United States |
| Cook Children's Health Care System | Fort Worth | Texas | 76104 | United States |
| University of Utah - Primary Children's Medical Center | Salt Lake City | Utah | 84132 | United States |
| Vermont Lung Center | Colchester | Vermont | 05446 | United States |
| University of Virginia Health System | Charlottesville | Virginia | 22908 | United States |
| Children's Hospital of the King's Daughters | Norfolk | Virginia | 23507 | United States |
| University of Washington Medical Center | Seattle | Washington | 98195 | United States |
| Providence Pediatric Pulmonary & Cystic Fibrosis Clinic | Spokane | Washington | 99204 | United States |
| West Virginia University | Morgantown | West Virginia | 26506 | United States |
| University Hospital and UW Health Clinics | Madison | Wisconsin | 53792 | United States |
| Royal Prince Alfred Hospital | Camperdown | Australia |
| The Prince Charles Hospital | Chermside | Australia |
| Alfred Hospital | Melbourne, VIC | Australia |
| Institute for Respiratory Health | Nedlands | Australia |
| Telethon Kids Institute | Nedlands | Australia |
| The Royal Children's Hospital | Parkville, VIC | Australia |
| Sydney Children's Hospital | Randwick | Australia |
| Mater Adult Hospital | South Brisbane | Australia |
| Queensland Children's Hospital | South Brisbane | Australia |
| Westmead Hospital | Westmead | Australia |
| University of Graz | Graz | Austria |
| Medizinische Universität Innsbruck | Innsbruck | Austria |
| Uniklinikum Salzburg - Universitätsklinik für Pneumologie/Lungenheilkunde | Salzburg | Austria |
| Medizinische Universität Wien | Vienna | Austria |
| Abteilung fuer Pulmologie Klinikum Wels-Grieskirchen | Wels | Austria |
| Cliniques Universitaires de Bruxelles Hopital Erasme | Brussels | Belgium |
| Universitair Ziekenhuis Brussel - Campus Jette | Brussels | Belgium |
| Universitair Ziekenhuis Gent | Ghent | Belgium |
| Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg | Leuven | Belgium |
| Cliniques Universitaires Saint-Luc | Woluwe-Saint-Lambert | Belgium |
| University of Calgary Medical Clinic of the Foothills Medical Centre | Calgary | Canada |
| Stollery Children's Hospital | Edmonton | Canada |
| St. Joseph's Health Care London | London | Canada |
| Centre Hospitalier de l'Universite de Montreal (CHUM) Hotel-Dieu | Montreal | Canada |
| McGill University Health Centre, Glen Site, Montreal Children's Hospital | Montreal | Canada |
| Institut Universitaire de Cardiologie et Pneumologie de Quebec - Universite Laval | Québec | Canada |
| St. Michael's Hospital | Toronto | Canada |
| British Columbia Children's Hospital | Vancouver | Canada |
| St. Paul's Hospital | Vancouver | Canada |
| Aarhus University Hospital | Aarhus | Denmark |
| Juliane Marie Center, Rigshospitalet | Copenhagen | Denmark |
| Groupe Hospitaler Pellegrin, CHU De Bordeaux | Bordeaux | France |
| CHU Lyon - Hopital Femme Mere-Enfant | Bron | France |
| Centre Hospitalier Intercommunal Creteil | Créteil | France |
| Institut Cœur Poumon, CHU de Lille | Lille | France |
| Hopital Arnaud de Villeneuve | Montpellier | France |
| Centre Hospitalier Universitaire De Nantes - G. R. Laennec | Nantes | France |
| Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur | Nice | France |
| Hopital Cochin | Paris | France |
| Hopital Necker, Enfants Malades | Paris | France |
| Hopital Robert Debre | Paris | France |
| Centre Hospitalier Lyon Sud | Pierre-Bénite | France |
| Centre de Perharidy | Roscoff | France |
| CHU de Rouen - Hopital Charles Nicolle | Rouen Cedex, Seine Maritime | France |
| Hôpital de Hautepierre, AX5 | Strasbourg | France |
| Hopital Foch (Suresnes), Hopital Foch, Adultes | Suresnes | France |
| Hopital Bretonneau | Tours | France |
| Centre hospitalier universitaire de Nancy, Hôpital de Braboi | Vandœuvre-lès-Nancy | France |
| Charite Paediatric Pulmonology Department | Berlin | Germany |
| Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital | Erlangen | Germany |
| Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen | Essen | Germany |
| Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie | Essen | Germany |
| Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin | Giessen | Germany |
| Pneumologisches Studienzentrum Muenchen-West | München | Germany |
| General Hospital of Thessaloniki "Papanikolaou" | Pilea Chortiatis | Greece |
| General Hospital of Thessaloniki 'Hippokratio', 3rd Pediatrics Department of Aristotle University of Thessaloniki | Thessaloniki | Greece |
| National Koranyi Institute for TBC and Pulmonology | Budapest | Hungary |
| Pulmonology Institute Torokbalint | Törökbálint | Hungary |
| Cork University Hospital | Cork | Ireland |
| Children's Health Ireland at Crumlin | Dublin | Ireland |
| Children's Health Ireland at Tallaght | Dublin | Ireland |
| Children's University Hospital, Dublin | Dublin | Ireland |
| St. Vincent's University Hospital | Dublin | Ireland |
| University Hospital Limerick (Adults) | Limerick | Ireland |
| University Hospital Limerick (Pediatrics) | Limerick | Ireland |
| Hadassah Medical Organization | Jerusalem | Israel |
| Schneider Children's Medical Center of Israel | Petach Tikvah | Israel |
| Sheba Medical Center - The Edmond and Lili Safra Children's Hospital | Tel Litwinsky | Israel |
| Azienda Ospedaliero Universitaria Ospedale Riuniti | Ancona | Italy |
| Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer | Florence | Italy |
| IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico | Genova | Italy |
| Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena | Milan | Italy |
| Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico | Milan | Italy |
| Malattie Apparato Respiratorio 2 - Centro Fibrosi Cistica | Orbassano | Italy |
| Centro Regionale Fibrosi Cistica, A.O. Ospedale San Carlo | Potenza | Italy |
| Azienda Ospedaliera di Verona - Ospedale Civile Maggiore | Verona | Italy |
| Academisch Medisch Centrum (Academic Medical Centre) | Amsterdam | Netherlands |
| UMC St. Radboud | Nijmegen | Netherlands |
| Erasmus Medical Center | Rotterdam | Netherlands |
| HagaZiekenhuis van den Haag | The Hague | Netherlands |
| University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis | Utrecht | Netherlands |
| Greenlane Clinical Centre | Auckland | New Zealand |
| Starship Children's Hospital | Auckland | New Zealand |
| Canterbury Respiratory Research Group | Christchurch Hospital | New Zealand |
| Waikato Hospital | Hamilton | New Zealand |
| Haukeland Universitetssjukehus (CF) | Bergen | Norway |
| Oslo University Hospital, Department of Paediatric Medicine | Oslo | Norway |
| Pediatric Hospital Polanki named of Maciej Płażyński | Gdansk | Poland |
| Institute of Tuberculosis and Lung Diseases | Rabka-Zdrój | Poland |
| Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY | Łomianki | Poland |
| Sahlgrenska Universitetssjukhuset | Gothenburg | Sweden |
| Lund University Skanes Universitetssjukhus | Malmö | Sweden |
| Karolinska Universitetssjukhuset, Huddinge | Stockholm | Sweden |
| Lindenhofspital - Quartier Bleu | Bern | Switzerland |
| Réseau Hospitalier Neuchâtelois | Neuchâtel | Switzerland |
| Kinderspital Zuerich | Zurich | Switzerland |
| Birmingham Children's Hospital | Birmingham | United Kingdom |
| Royal Papworth Hospital NHS Foundation Trust | Cambridge | United Kingdom |
| Clinical Research Facility, Queen Elizabeth University Hospital | Glasgow | United Kingdom |
| Leeds General Infirmary | Leeds | United Kingdom |
| St. James University Hospital | Leeds | United Kingdom |
| King's College Hospital | London | United Kingdom |
| Royal Brompton Hospital | London | United Kingdom |
| St. Bartholomew's Hospital | London | United Kingdom |
| Royal Manchester Children's Hospital | Manchester | United Kingdom |
| Wythenshawe Hospital | Manchester | United Kingdom |
| All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough | Penarth | 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. |
Following ELX/TEZ/IVA run-in period of 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks. |
| Pooled Analysis Set | The Pooled Full Analysis Set (PFAS) included all randomized participants from this study (VX20-121-103) and from Study VX20-121-102 who carry the intended CFTR genotype and received at least 1dose of study drug during the Treatment Period. |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
All participants who received at least 1 dose of study drug during the treatment Period.
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| ID | Title | Description |
|---|---|---|
| BG000 | ELX/TEZ/IVA | Following ELX/TEZ/IVA run-in period of 4 weeks, participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 52 weeks. |
| BG001 | VX-121/TEZ/D-IVA | Following ELX/TEZ/IVA run-in period of 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks. |
| BG002 | Total | Total of all reporting groups |
| 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 |
| |||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants | No |
| |||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants | No |
| |||||||||||||||
| Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | Here, "Number Analyzed" signifies participants who were evaluable for this study specific baseline measure. This analysis set (N=565) included participants who received the dose and who had a data for this efficacy analysis. | Mean | Standard Deviation | Percentage points |
|
| 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 | Absolute Change in Percent Predicted Forced Expiratory Volume in 1second (ppFEV1) | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | The Full Analysis Set (FAS) included all randomized participants who carried the intended CFTR mutation(s) and received at least 1 dose of study drug during the Treatment Period. Here "Overall Number of participants Analyzed" signifies those participants who were evaluated for this specific outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | percentage points | From Baseline Through Week 24 |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in Sweat Chloride (SwCl) | Sweat samples were collected using an approved collection device. | FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this specific outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | millimole per liter (mmol/L) | From Baseline Through Week 24 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With SwCl <60 Millimole Per Liter (mmol/L) (Pooled With Data From Study VX20-121-102) | Sweat samples were collected using an approved collection device. | The Pooled Full Analysis Set (PFAS) included all randomized participants from this study (VX20-121-102) and from Study VX20-121-103 who carried the intended CFTR mutation(s) and received at least 1 dose of study drug during the Treatment Period. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this specific outcome measure. | Posted | Number | percentage of participants | From Baseline Through Week 24 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With SwCl <30 mmol/L (Pooled With Data From Study VX20-121-102) | Sweat samples were collected using an approved collection device. | PFAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this specific outcome measure. | Posted | Number | percentage of participants | From Baseline Through Week 24 |
|
|
Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
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 | ELX/TEZ/IVA | Following ELX/TEZ/IVA run-in period of 4 weeks, subjects received ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 52 weeks. | 0 | 289 | 40 | 289 | 253 | 289 |
| EG001 | VX-121/TEZ/D-IVA | Following ELX/TEZ/IVA run-in period of 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks. | 0 | 284 | 40 | 284 | 252 | 284 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Leukocytosis | Blood and lymphatic system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Vision blurred | Eye disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Distal intestinal obstruction syndrome | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Faecaloma | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Melaena | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Pancreatic cyst | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Cholangitis | Hepatobiliary disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Fatty liver alcoholic | Hepatobiliary disorders | MedDRA 26.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Erysipelas | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Infective exacerbation of bronchiectasis | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Lower respiratory tract infection bacterial | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Metapneumovirus pneumonia | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Pyelonephritis | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Respiratory tract infection viral | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Ankle fracture | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Craniocerebral injury | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Hand fracture | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Post procedural haematoma | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Skin laceration | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Skull fractured base | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 26.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 26.1 | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA 26.1 | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA 26.1 | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA 26.1 | Systematic Assessment |
| |
| Glycosylated haemoglobin increased | Investigations | MedDRA 26.1 | Systematic Assessment |
| |
| Malnutrition | Metabolism and nutrition disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Type 3 diabetes mellitus | Metabolism and nutrition disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Disturbance in attention | Nervous system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Haemorrhage intracranial | Nervous system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Post-traumatic headache | Nervous system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Psychomotor hyperactivity | Nervous system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Device leakage | Product Issues | MedDRA 26.1 | Systematic Assessment |
| |
| Acute stress disorder | Psychiatric disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Suicidal ideation | Psychiatric disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Intermenstrual bleeding | Reproductive system and breast disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Vaginal cyst | Reproductive system and breast disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 26.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 26.1 | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA 26.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 26.1 | 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 | Sep 19, 2023 | May 10, 2024 | 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 Hispanic or Latino |
|
|
| Not Collected per Local Regulations |
|
|
|
| Asian |
|
|
| American Indian or Alaska Native |
|
|
| Other |
|
|
| Not Collected per Local Regulations |
|
|
| More than one race |
|
|
|
|
|
|
|
|
|