Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy
- Registration Number
- NCT04058366
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
This study evaluated the long-term safety, efficacy, 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 the F508del mutation and a gating or residual function mutation (F/G and F/RF genotypes).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 251
- Completed study drug treatment in parent study (VX18-445-104); or had study drug interruption(s) in parent study but completed study visits up to the last scheduled visit of the Treatment Period in the parent study
Key
- History of study drug intolerance in parent study
Other protocol defined Inclusion/Exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description ELX/TEZ/IVA ELX/TEZ/IVA Part A: Participants received ELX (elexacaftor) 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 96 weeks. Part B: Participants from certain countries participated in Part B and continued to receive ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks. ELX/TEZ/IVA IVA Part A: Participants received ELX (elexacaftor) 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 96 weeks. Part B: Participants from certain countries participated in Part B and continued to receive ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks.
- Primary Outcome Measures
Name Time Method Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) From Baseline up to Week 100
- Secondary Outcome Measures
Name Time Method Part A: Absolute Change From Parent Study Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score From Baseline at Week 96 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.
Part A: Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl) From Baseline at Week 96 Sweat samples were collected using an approved collection device.
Part A: Absolute Change From Parent Study Baseline in Body Mass Index (BMI) From Baseline at Week 96 BMI was defined as weight in kilogram (kg) divided by height in square meter (m\^2).
Part A: Absolute Change From Parent Study Baseline in BMI Z-score From Baseline at Week 96 BMI was defined as weight in kilogram (kg) divided by squared height in meters (m\^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard.
Part A: Absolute Change From Parent Study Baseline in Body Weight From Baseline at Week 96 Part A: Absolute Change From Parent Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) From Baseline at Week 96 FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Trial Locations
- Locations (84)
Banner University of Arizona Medical Center
🇺🇸Tucson, Arizona, United States
Miller Children's Hospital / Long Beach Memorial
🇺🇸Long Beach, California, United States
Stanford University
🇺🇸Palo Alto, California, United States
University of California Davis Medical Center
🇺🇸Sacramento, California, United States
University of California San Francisco, Lung Transplant Program
🇺🇸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
Scroll for more (74 remaining)Banner University of Arizona Medical Center🇺🇸Tucson, Arizona, United States