Cystic Fibrosis Clinical Trial
Official title:
A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)
| Verified date | June 2021 |
| Source | Vertex Pharmaceuticals Incorporated |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
This study will evaluate the efficacy, safety and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a gating or residual function mutation (F/G and F/RF genotypes).
| Status | Completed |
| Enrollment | 271 |
| Est. completion date | June 12, 2020 |
| Est. primary completion date | June 12, 2020 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 12 Years and older |
| Eligibility | Key Inclusion Criteria: - Subject has a confirmed diagnosis of CF and is heterozygous for F508del and either a gating or residual function mutation (F/G and F/RF genotypes) - Forced expiratory volume in 1 second (FEV1) value =40% and =90% of predicted mean for age, sex, and height Key Exclusion Criteria: - Clinically significant cirrhosis with or without portal hypertension - Lung infection with organisms associated with a more rapid decline in pulmonary status - Solid organ or hematological transplantation Other protocol defined Inclusion/Exclusion criteria may apply |
| Country | Name | City | State |
|---|---|---|---|
| Australia | The Prince Charles Hospital | Chermside | |
| Australia | Alfred Hospital | Melbourne, VIC | |
| Australia | Perth Children's Hospital | Nedlands | |
| Australia | The Royal Children's Hospital | Parkville, VIC | |
| Australia | Mater Adult Hospital | South Brisbane | |
| Australia | Queensland Children's Hospital | South Brisbane | |
| Australia | Westmead Hospital | Westmead | |
| Belgium | Cliniques Universitaires de Bruxelles Hopital Erasme | Brussels | |
| Belgium | Universitair Ziekenhuis Brussel - Campus Jette | Brussels | |
| Belgium | Universitair Ziekenhuis Gent | Gent | |
| Belgium | Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg | Leuven | |
| Canada | Stollery Children's Hospital | Edmonton | |
| Canada | McGill University Health Center | Québec | |
| Canada | St. Michael's Hospital | Toronto | |
| Canada | St. Paul's Hospital | Vancouver | |
| Denmark | Juliane Marie Center, Rigshospitalet | Copenhagen | |
| France | Centre Hospitalier Lyon Sud | Benite Cedex | |
| France | Groupe Hospitaler Pellegrin, CHU De Bordeaux | Bordeaux cedex | |
| France | CHRU de Lille - Hopital Albert Calmette | Lille | |
| France | CHU Marseille - Hopital Nord | Marseille | |
| France | CHU de Montpellier - Hopital Arnaud de Villeneuve | Montpellier Cedex 5 | |
| France | Hopital Cochin | Paris | |
| France | Hopital Necker, Enfants Malades | Paris Cedex 15 | |
| France | Hopital Pontchaillou CHU de Rennes | Rennes Cedex | |
| Germany | 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 | Justus-Liebig-Universitaet Gießen Zentrum fur Kinderheilkunde und Jugendmedizin | Giessen | |
| Germany | Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie | Halle | |
| Germany | Pneumologisches Studienzentrum Muenchen-West | Muenchen | |
| Germany | University Hospital Wuerzburg | Würzburg | |
| Ireland | Cork University Hospital | Cork | |
| Ireland | Beaumont Hospital | Dublin | |
| Ireland | St. Vincent's University Hospital | Dublin | |
| Israel | University Hospital Limerick (Adults) | Limerick | |
| Italy | Azienda Ospedaliero Universitaria Ospedale Riuniti | Ancona | |
| Italy | IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico | Genova | |
| Italy | Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico | Milano | |
| Italy | Malattie Apparato Respiratorio 2 - Fibrosi Cistica | Orbassano | |
| Italy | Ospedale Pediatrico Bambino Gesu | Rome | |
| Italy | Azienda Ospedaliera di Verona-Ospedale Civile Maggiore | Verona | |
| Netherlands | HagaZiekenhuis van den Haag | Den Haag | |
| Netherlands | University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis | Heidelberglaan | |
| Netherlands | UMC St. Radboud | Nijmegen | |
| Spain | Hospital Universitari Vall d´Hebron Servicio de Broncoscopia | Barcelona | |
| Spain | Hospital Virgen de la Arrixaca | Murcia | |
| Spain | Hospital Universitario Virgen del Rocio | Sevilla | |
| Spain | Hospital Universitario y Politecnico La Fe | Valencia | |
| United Kingdom | Papworth Hospital NHS Foundation Trust, Papworth Everard | Cambridge | |
| United Kingdom | Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital | Exeter | |
| United Kingdom | Clinical Research Facility, Queen Elizabeth University 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 | Southampton General Hospital | Southampton | |
| United States | Michigan Medicine | Ann Arbor | Michigan |
| United States | Dell Children's Medical Group | Austin | Texas |
| United States | University of Alabama at Birmingham | Birmingham | Alabama |
| United States | Boston Children's Hospital | Boston | Massachusetts |
| United States | Massachusetts General Hospital Cystic Fibrosis Center Clinical Rsearch Center | Boston | Massachusetts |
| United States | University of North Carolina Hospitals | Chapel Hill | North Carolina |
| United States | UC Health Holmes | Cincinnati | Ohio |
| United States | Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center | Cleveland | Ohio |
| United States | Nationwide Children's Hospital | Columbus | Ohio |
| United States | The University of Texas Southwestern Medical Center | Dallas | Texas |
| United States | National Jewish Health | Denver | Colorado |
| United States | Texas Children's Hospital | Houston | Texas |
| United States | Indiana University | Indianapolis | Indiana |
| United States | The University of Iowa Hospitals and Clinics | Iowa City | Iowa |
| United States | University of Kansas Medical Center | Kansas City | Kansas |
| United States | Dartmouth Hitchcock Medical Center | Lebanon | New Hampshire |
| United States | University of Kentucky | Lexington | Kentucky |
| United States | Miller Children's Hospital / Long Beach Memorial | Long Beach | California |
| United States | University of Wisconsin Hospital and Clinics | Madison | Wisconsin |
| United States | University of Miami Miller School of Medicine | Miami | Florida |
| United States | University of Minnesota | Minneapolis | Minnesota |
| United States | Northwell Health- Long Island Jewish Medical Center | New Hyde Park | New York |
| United States | Columbia University Medical Center | New York | New York |
| United States | Mount Sinai Beth Israel | New York | New York |
| United States | University of Oklahoma Health Sciences Center | Oklahoma City | Oklahoma |
| United States | Central Florida Pulmonary Group, PA | Orlando | Florida |
| United States | Stanford University | Palo Alto | California |
| United States | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania |
| United States | Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania |
| United States | Oregon Health & Science University | Portland | Oregon |
| United States | University of California Davis Medical Center | Sacramento | California |
| United States | Washington University School of Medicine / St. Louis Children's Hospital | Saint Louis | Missouri |
| United States | University of Utah / Primary Children's Medical Center | Salt Lake City | Utah |
| United States | University of California San Francisco, Lung Transplant Program | San Francisco | California |
| United States | University of Washington Medical Center | Seattle | Washington |
| United States | ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center | Toledo | Ohio |
| United States | Banner University of Arizona Medical Center | Tucson | Arizona |
| Lead Sponsor | Collaborator |
|---|---|
| Vertex Pharmaceuticals Incorporated |
United States, Australia, Belgium, Canada, Denmark, France, Germany, Ireland, Israel, Italy, Netherlands, Spain, United Kingdom,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for ELX/TEZ/IVA Group | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | From Baseline Through Week 8 | |
| Secondary | Absolute Change in Sweat Chloride (SwCl) for ELX/TEZ/IVA Group | Sweat samples were collected using an approved collection device. | From Baseline Through Week 8 | |
| Secondary | Absolute Change in ppFEV1 for the ELX/TEZ/IVA Group Compared to the Control Group | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | From Baseline Through Week 8 | |
| Secondary | Absolute Change in SwCl for ELX/TEZ/IVA Group Compared to the Control Group | Sweat samples were collected using an approved collection device. | From Baseline Through Week 8 | |
| Secondary | Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for ELX/TEZ/IVA Group | 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. | From Baseline Through Week 8 | |
| Secondary | Absolute Change in CFQ-R Respiratory Domain Score for ELX/TEZ/IVA Group Compared to the Control Group | 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. | From Baseline Through Week 8 | |
| Secondary | Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Day 1 up to Week 12 |
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