Cystic Fibrosis Clinical Trial
Official title:
A Phase 2, Randomized, Double-blind Study to Evaluate the Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis
| Verified date | December 2021 |
| Source | Vertex Pharmaceuticals Incorporated |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
The purpose of this study is to evaluate the efficacy, safety, pharmacodynamic (PD) and pharmacokinetic (PK) effect of VX-561.
| Status | Completed |
| Enrollment | 77 |
| Est. completion date | August 20, 2020 |
| Est. primary completion date | August 20, 2020 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years and older |
| Eligibility | Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value =40% and =100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply |
| Country | Name | City | State |
|---|---|---|---|
| Australia | The Prince Charles Hospital | Chermside | |
| Australia | Alfred Hospital | Melbourne, VIC | |
| Australia | Mater Adult Hospital | South Brisbane | |
| Australia | Westmead Hospital | Westmead | |
| Belgium | Universitair Ziekenhuis Gent | Gent | |
| Germany | Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital | Erlangen | |
| Germany | Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen | Essen | |
| 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 | St. Vincent's University Hospital | Dublin | |
| Ireland | Cork University Hospital | Dublin 12 | |
| Ireland | Beaumont Hospital | Dublin 9 | |
| Ireland | University Hospital Limerick (Adults) | Limerick | |
| Netherlands | UMC St. Radboud | Nijmegen | |
| 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 States | Michigan Medicine | Ann Arbor | Michigan |
| United States | Dell Children's Medical Group | Austin | Texas |
| United States | Johns Hopkins Hospital | Baltimore | Maryland |
| 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 Research Center | Boston | Massachusetts |
| United States | University of North Carolina Hospitals | Chapel Hill | North Carolina |
| United States | Medical University of South Carolina | Charleston | South Carolina |
| United States | UC Health Holmes | Cincinnati | Ohio |
| United States | The University of Texas Southwestern Medical Center | Dallas | Texas |
| United States | National Jewish Health | Denver | Colorado |
| United States | Indiana University | Indianapolis | Indiana |
| 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 | Columbia University Medical Center | 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 | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania |
| United States | Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania |
| United States | Cardinal Glennon Children's Hospital | Saint Louis | Missouri |
| 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 | UCSF Gateway Medical Center | San Francisco | California |
| 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, Germany, Ireland, 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 12 | |
| Secondary | Absolute Change in Sweat Chloride (SwCl) | Sweat samples were collected using an approved collection device. | From Baseline at Week 12 | |
| Secondary | Observed Pre-Dose Concentration (Ctrough) of VX-561 and Its Metabolites (M1-VX-561 and M6-VX-561) and IVA and Its Metabolites (M1-IVA and M6-IVA) | At Week 4 | ||
| Secondary | Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Baseline up to Week 16 |
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