Evaluation of ELX/TEZ/IVA in Cystic Fibrosis (CF) Subjects 2 Through 5 Years
- Registration Number
- NCT04537793
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
This study will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) triple combination therapy in CF subjects 2 through 5 years of age.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 83
- Homozygous for the F508del mutation or heterozygous for F508del and a minimal function (MF) mutation (F/F or F/MF genotypes)
Key
- Clinically significant cirrhosis with or without portal hypertension
- Lung infection with organisms associated with a more rapid decline in pulmonary status
- Solid organ or hematological transplantation
Other protocol defined Inclusion/Exclusion criteria may apply
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Part A: ELX/TEZ/IVA IVA Participants weighing greater than or equal to (\>=)14 kilograms (kg) at screening received elexacaftor (ELX) 100 milligrams (mg) once daily (qd)/tezacaftor (TEZ) 50 mg qd/ivacaftor (IVA) 75 mg every 12 hours (q12h) in the treatment period for 15 days. Part B: ELX/TEZ/IVA IVA Participants weighing (\>=)14 kg at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h. Participants weighing (\>=)10 kg to less than (\<)14 kg received ELX 80 mg qd/TEZ 40 mg qd/IVA 60 mg once every morning (qAM) and 59.5 mg once every evening (qPM) in the treatment period for 24 weeks. Part A: ELX/TEZ/IVA ELX/TEZ/IVA Participants weighing greater than or equal to (\>=)14 kilograms (kg) at screening received elexacaftor (ELX) 100 milligrams (mg) once daily (qd)/tezacaftor (TEZ) 50 mg qd/ivacaftor (IVA) 75 mg every 12 hours (q12h) in the treatment period for 15 days. Part B: ELX/TEZ/IVA ELX/TEZ/IVA Participants weighing (\>=)14 kg at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h. Participants weighing (\>=)10 kg to less than (\<)14 kg received ELX 80 mg qd/TEZ 40 mg qd/IVA 60 mg once every morning (qAM) and 59.5 mg once every evening (qPM) in the treatment period for 24 weeks.
- Primary Outcome Measures
Name Time Method Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites From Day 1 through Day 15 Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) From Day 1 up to Day 43 Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) From Day 1 up to Week 28
- Secondary Outcome Measures
Name Time Method Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites From Day 1 through Week 16 Part B: Absolute Change in Sweat Chloride (SwCl) From Baseline through Week 24 Sweat samples were collected using an approved collection device.
Part B: Absolute Change in Lung Clearance Index 2.5 (LCI 2.5) From Baseline through Week 24 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.
Trial Locations
- Locations (22)
UPMC Children's Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Banner University of Arizona Medical Center
🇺🇸Tucson, Arizona, United States
Stanford University
🇺🇸Palo Alto, California, United States
Oregon Health & Science University
🇺🇸Portland, Oregon, United States
Children's Hospital of Colorado
🇺🇸Aurora, Colorado, United States
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Riley Hospital for Children at Indiana University Health
🇺🇸Indianapolis, Indiana, United States
The Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
British Columbia Children's Hospital
🇨🇦Vancouver, Canada
The Royal Children's Hospital
🇦🇺Parkville, VIC, Australia
Queensland Children's Hospital
🇦🇺South Brisbane, Australia
NC TraCS Institute - CTRC University of North Carolina at Chapel Hill
🇺🇸Chapel Hill, North Carolina, United States
Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie
🇩🇪Essen, Germany
Charite Paediatric Pulmonology Department
🇩🇪Berlin, Germany
Alder Hey Children's NHS Foundation Trust
🇬🇧Liverpool, United Kingdom
Washington University School of Medicine / St. Louis Children's Hospital
🇺🇸Saint Louis, Missouri, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
The Hospital for Sick Children
🇨🇦Toronto, Canada
Telethon Kids Institute
🇦🇺Nedlands, Australia
Royal Brompton Hospital
🇬🇧London, United Kingdom