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 Homozygous for the F508del Mutation (F/F)
| Verified date | January 2020 |
| Source | Vertex Pharmaceuticals Incorporated |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
This study will evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation (F/F)
| Status | Completed |
| Enrollment | 113 |
| Est. completion date | December 28, 2018 |
| Est. primary completion date | December 28, 2018 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 12 Years and older |
| Eligibility |
Key Inclusion Criteria: - Homozygous for the F508del mutation (F/F) - 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 |
|---|---|---|---|
| Belgium | Cliniques Universitaires de Bruxelles Hopital Erasme | Brussels | |
| Belgium | Universitair Ziekenhuis Brussel - Campus Jette | Brussels | |
| Belgium | UZ Antwerpen | Edegem | |
| Belgium | Universitair Ziekenhuis Gent | Gent | |
| Belgium | Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg | Leuven | |
| Netherlands | Academic Medical Center | Amsterdam | |
| Netherlands | HagaZiekenhuis van den Haag | Den Haag | |
| Netherlands | University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis | Heidelberglaan | |
| Netherlands | UMC St. Radboud | Nijmegen | |
| Netherlands | Erasmus Medical Center | Rotterdam | |
| United Kingdom | The Royal Belfast Hospital for Sick Children | Belfast | |
| United Kingdom | Heart of England NHS Foundation Trust, Birmingham Heartlands Hospital | Birmingham | |
| United Kingdom | Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital | Exeter | |
| United Kingdom | Leeds General Infirmary | Leeds | |
| United Kingdom | King's College Hospital | London | |
| United Kingdom | Southampton General Hospital | Southampton | |
| United States | Massachusetts General Hospital Cystic Fibrosis Center Clinical Research Center | Boston | Massachusetts |
| United States | UNC Marisco Clinical Research Center | Chapel Hill | North Carolina |
| United States | Medical University of South Carolina | Charleston | South Carolina |
| United States | University of Virginia Primary Care Center | Charlottesville | Virginia |
| United States | Northwestern Memorial Hospital | Chicago | Illinois |
| United States | Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center | Cleveland | Ohio |
| United States | Vermont Lung Center | Colchester | Vermont |
| United States | Nationwide Children's Hospital | Columbus | Ohio |
| United States | The University of Texas Southwestern Medical Center | Dallas | Texas |
| United States | Dayton Children's Hospital | Dayton | Ohio |
| United States | National Jewish Health | Denver | Colorado |
| United States | Harper University Hospital | Detroit | Michigan |
| United States | Joe DiMaggio Cystic Fibrosis & Pulmonary Center/ Joe DiMaggio Children's Hospital/ Memorial Regional Hospital | Hollywood | Florida |
| United States | University of Arkansas for Medical Sciences | Little Rock | Arkansas |
| United States | Miller Children's Hospital/ Long Beach Memorial | Long Beach | California |
| United States | Children's Hospital Los Angeles | Los Angeles | California |
| United States | Valley Children's Hospital | Madera | California |
| United States | West Virginia University | Morgantown | West Virginia |
| United States | Tulane Medical Center | New Orleans | Louisiana |
| United States | University of Oklahoma Health Sciences Center | Oklahoma City | Oklahoma |
| United States | Central Florida Pulmonary Group, PA | Orlando | Florida |
| United States | Nemours Children's Hospital | Orlando | Florida |
| United States | Saint Francis Medical Center/ Children's Hospital of Illinoise/OSF | Peoria | Illinois |
| United States | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania |
| United States | Tampa General Hospital Cardiac and Lung Transplant Clinic | Tampa | Florida |
| 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 |
| United States | New York Medical College | Valhalla | New York |
| Lead Sponsor | Collaborator |
|---|---|
| Vertex Pharmaceuticals Incorporated |
United States, Belgium, Netherlands, United Kingdom,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Absolute Change in 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. | From Baseline at Week 4 | |
| Secondary | Absolute Change in Sweat Chloride (SwCl) | Sweat samples were collected using an approved collection device. | From Baseline at Week 4 | |
| Secondary | Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score | 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 indicate fewer symptoms and better health-related quality of life. | From Baseline at Week 4 | |
| Secondary | Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | From first dose of study drug in TC treatment period up to 28 days after last dose of study drug or to the completion of study participation date, whichever occurs first (up to Week 8) | ||
| Secondary | Observed Pre-Dose Concentration (Ctrough) of VX-445, TEZ, TEZ Metabolite (M1-TEZ), and IVA | Day 1 and Week 4 |
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