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)
University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
Banner University of Arizona Medical Center
🇺🇸Tucson, Arizona, United States
Miller Children's Hospital / Long Beach Memorial
🇺🇸Long Beach, California, United States
UCSF Gateway Medical Center
🇺🇸San Francisco, California, United States
National Jewish Health
🇺🇸Denver, Colorado, United States
University of Miami Miller School of Medicine
🇺🇸Miami, Florida, United States
Central Florida Pulmonary Group, PA
🇺🇸Orlando, Florida, United States
Indiana University
🇺🇸Indianapolis, Indiana, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States
University of Kentucky
🇺🇸Lexington, Kentucky, United States
Scroll for more (38 remaining)University of Alabama at Birmingham🇺🇸Birmingham, Alabama, United States