Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA TC Combination Therapy in Participants With Cystic Fibrosis Who Are 6 Years of Age and Older
- Registration Number
- NCT04183790
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
The study evaluates the long-term safety, tolerability, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in participants with cystic fibrosis (CF).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 64
- Completed study drug treatment in parent study (VX18-445-106 Part B, NCT03691779), 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 Participants greater than or equal to (≥) 6 years and less than (\<) 12 years of age and weighing \<30 kilograms (kg) received ELX (elexacaftor) 100 milligram (mg) once daily (qd) /TEZ (tezacaftor) 50 mg qd/IVA (ivacaftor) 75 mg every 12 hours (q12h) and those weighing (≥) 30 kg received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg in the treatment period for up to 192 weeks. Participants ≥12 years of age received ELX 200 mg qd/ TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 192 weeks. ELX/TEZ/IVA IVA Participants greater than or equal to (≥) 6 years and less than (\<) 12 years of age and weighing \<30 kilograms (kg) received ELX (elexacaftor) 100 milligram (mg) once daily (qd) /TEZ (tezacaftor) 50 mg qd/IVA (ivacaftor) 75 mg every 12 hours (q12h) and those weighing (≥) 30 kg received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg in the treatment period for up to 192 weeks. Participants ≥12 years of age received ELX 200 mg qd/ TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 192 weeks.
- Primary Outcome Measures
Name Time Method 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 196
- Secondary Outcome Measures
Name Time Method Number of Participants With Pulmonary Exacerbations (PEx) for 106/107 From Baseline up to Week 192 Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
Number of CF-related Hospitalizations for 106/107 From Baseline up to Week 192 The total number of CF related hospitalization (Planned + Unplanned) events across all participants were reported.
Absolute Change in BMI-for-age Z-score From Baseline up to Week 192 BMI was defined as weight in kg divided by squared height in meters (m\^2). The z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) From Baseline up to Week 192 FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Absolute Change in Sweat Chloride (SwCl) From Baseline up to Week 192 Sweat samples were collected using an approved collection device.
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score From Baseline up to Week 192 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.
Absolute Change in Body Mass Index (BMI) From Baseline up to Week 192 BMI was defined as weight in kg divided by squared height in meters (m\^2).
Absolute Change in Lung Clearance Index 2.5 (LCI 2.5) From Baseline up to Week 192 The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.
Absolute Change in Weight From Baseline up to Week 192 Absolute Change in Weight-for-age Z-score From Baseline up to Week 192 The z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
Absolute Change in Height From Baseline up to Week 192 Absolute Change in Height-for-age Z-score From Baseline up to Week 192 The z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
Trial Locations
- Locations (21)
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
Ann & Robert Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
Texas Children's Hospital
🇺🇸Houston, Texas, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
The Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
Northwell Health- Long Island Jewish Medical Center
🇺🇸New Hyde Park, New York, United States
Clinical Research of Charlotte
🇺🇸Charlotte, North Carolina, United States
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
🇺🇸Cleveland, Ohio, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Oregon Health & Science University
🇺🇸Portland, Oregon, United States
The Children's Hospital at Westmead
🇦🇺Westmead, Australia
Queensland Children's Hospital
🇦🇺South Brisbane, Australia
The Hospital for Sick Children
🇨🇦Toronto, Canada
British Columbia's Children's Hospital
🇨🇦Vancouver, Canada
Children's Health Ireland at Temple Street
🇮🇪Dublin, Ireland
Birmingham Children's Hospital - NHS Foundation Trust
🇬🇧Birmingham, United Kingdom
Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
🇬🇧London, United Kingdom
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Children's Hospital of Orange County
🇺🇸Orange, California, United States
Children's Health Ireland at Crumlin
🇮🇪Dublin, Ireland