A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis
- Registration Number
- NCT03911713
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
The purpose of this study is to evaluate the efficacy, safety, pharmacodynamic (PD) and pharmacokinetic (PK) effect of VX-561.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 77
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description VX-561: 250 mg Placebo Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks. VX-561: 150 mg Placebo Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks. VX-561: 25 mg Placebo Participants received VX-561 25 mg orally daily (qd) in the treatment period for 12 weeks. VX-561: 250 mg VX-561 Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks. Ivacaftor IVA Participants received IVA 150 milligrams (mg) orally every 12 hours (q12h) in the treatment period for 12 weeks. Ivacaftor Placebo Participants received IVA 150 milligrams (mg) orally every 12 hours (q12h) in the treatment period for 12 weeks. VX-561: 50 mg VX-561 Participants received VX-561 50 mg orally qd in the treatment period for 12 weeks. VX-561: 50 mg Placebo Participants received VX-561 50 mg orally qd in the treatment period for 12 weeks. VX-561: 25 mg VX-561 Participants received VX-561 25 mg orally daily (qd) in the treatment period for 12 weeks. VX-561: 150 mg VX-561 Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.
- Primary Outcome Measures
Name Time Method Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) From Baseline at Week 12 FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
- Secondary Outcome Measures
Name Time Method Absolute Change in Sweat Chloride (SwCl) From Baseline at Week 12 Sweat samples were collected using an approved collection device.
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 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
Trial Locations
- Locations (48)
Indiana University
🇺🇸Indianapolis, Indiana, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh of UPMC
🇺🇸Pittsburgh, Pennsylvania, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
University of Utah / Primary Children's Medical Center
🇺🇸Salt Lake City, Utah, United States
National Jewish Health
🇺🇸Denver, Colorado, United States
University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
Johns Hopkins Hospital
🇺🇸Baltimore, Maryland, 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
ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center
🇺🇸Toledo, Ohio, United States
University of Oklahoma Health Sciences Center
🇺🇸Oklahoma City, Oklahoma, United States
The Prince Charles Hospital
🇦🇺Chermside, Australia
Alfred Hospital
🇦🇺Melbourne, VIC, Australia
Westmead Hospital
🇦🇺Westmead, Australia
Mater Adult Hospital
🇦🇺South Brisbane, Australia
Universitair Ziekenhuis Gent
🇧🇪Gent, Belgium
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, Germany
Clinical Research Facility, Queen Elizabeth University Hospital
🇬🇧Glasgow, United Kingdom
St. James University Hospital
🇬🇧Leeds, United Kingdom
UCSF Gateway Medical Center
🇺🇸San Francisco, California, United States
University of Miami Miller School of Medicine
🇺🇸Miami, Florida, United States
Miller Children's Hospital / Long Beach Memorial
🇺🇸Long Beach, California, United States
Massachusetts General Hospital Cystic Fibrosis Center Clinical Research Center
🇺🇸Boston, Massachusetts, United States
University of Kentucky
🇺🇸Lexington, Kentucky, United States
Columbia University Medical Center
🇺🇸New York, New York, United States
UC Health Holmes
🇺🇸Cincinnati, Ohio, United States
University of Wisconsin Hospital and Clinics
🇺🇸Madison, Wisconsin, United States
Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital
🇩🇪Erlangen, Germany
Liverpool Heart and Chest Hospital
🇬🇧Liverpool, United Kingdom
UMC St. Radboud
🇳🇱Nijmegen, Netherlands
Banner University of Arizona Medical Center
🇺🇸Tucson, Arizona, United States
University Hospital Limerick (Adults)
🇮🇪Limerick, Ireland
St. Vincent's University Hospital
🇮🇪Dublin, Ireland
Beaumont Hospital
🇮🇪Dublin 9, Ireland
Dartmouth Hitchcock Medical Center
🇺🇸Lebanon, New Hampshire, United States
Cork University Hospital
🇮🇪Dublin 12, Ireland
The University of Texas Southwestern Medical Center
🇺🇸Dallas, Texas, United States
Central Florida Pulmonary Group, PA
🇺🇸Orlando, Florida, United States
Michigan Medicine
🇺🇸Ann Arbor, Michigan, United States
University of North Carolina Hospitals
🇺🇸Chapel Hill, North Carolina, United States
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
Dell Children's Medical Group
🇺🇸Austin, Texas, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States