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A Phase 3 Open-Label Study of ELX/TEZ/IVA in Children 6 Through 11 Years of Age With CF and at Least One F508del Allele.

  • Zemanick, Edith T1
  • Taylor-Cousar, Jennifer L2
  • Davies, Jane3
  • Gibson, Ronald L4
  • Mall, Marcus A5
  • McKone, Edward F6
  • McNally, Paul7
  • Ramsey, Bonnie W4
  • Rayment, Jonathan H8
  • Rowe, Steven M9
  • Tullis, Elizabeth10
  • Ahluwalia, Neil11
  • Chu, Chenghao11
  • Ho, Thang11
  • Moskowitz, Samuel M11
  • Noel, Sabrina11
  • Tian, Simon11
  • Waltz, David11
  • Weinstock, Tanya G11
  • Xuan, Fengjuan11
  • And 2 more
  • 1 University of Colorado Anschutz Medical Campus and Children's Hospital Colorado, Department of Pediatrics, Aurora, Colorado, United States. , (United States)
  • 2 National Jewish Health, Departments of Medicine and Pediatrics, Denver, Colorado, United States. , (United States)
  • 3 National Heart & Lung Institute; Imperial College London; NIHR Imperial Biomedical Research Centre and Royal Brompton & Harefield NHS Foundation Trust, London, United Kingdom of Great Britain and Northern Ireland. , (United Kingdom)
  • 4 University of Washington/Seattle Children's Hospital, Seattle, Washington, United States. , (United States)
  • 5 Charité-Universitätsmedizin Berlin; Berlin Institute of Health; and German Center for Lung Research, Department of Pediatric Pulmonology, Immunology and Intensive Care Medicine, Berlin, Germany. , (Germany)
  • 6 St Vincent's University Hospital & University College, Dublin 4, Ireland. , (Ireland)
  • 7 Children's Health Ireland and RCSI University of Medicine and Health Sciences, Dublin, Ireland. , (Ireland)
  • 8 British Columbia Children's Hospital, Vancouver, British Columbia, Canada. , (Canada)
  • 9 University of Alabama at Birmingham, Birmingham, Alabama, United States. , (United States)
  • 10 St Michael's Hospital, Toronto, Ontario, Canada. , (Canada)
  • 11 Vertex Pharmaceuticals Inc, 2184, Boston, Massachusetts, United States. , (United States)
  • 12 University of Queensland, Brisbane, Queensland, Australia. , (Australia)
  • 13 Ann & Robert H. Lurie Children's Hospital of Chicago, Pulmonary Medicine, Chicago, Illinois, United States. , (United States)
  • 14 Northwestern University Feinberg School of Medicine, Pediatrics, Chicago, Illinois, United States; [email protected] , (United States)
Published Article
American Journal of Respiratory and Critical Care Medicine
American Thoracic Society
Publication Date
Mar 18, 2021
DOI: 10.1164/rccm.202102-0509OC
PMID: 33734030


Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be efficacious and safe in patients aged 12 years and older with cystic fibrosis and at least one F508del-CFTR allele, but has not been evaluated in children <12 years of age. To assess the safety, pharmacokinetics, and efficacy of ELX/TEZ/IVA in children 6 through 11 years of age with F508del-minimal function or F508del-F508del genotypes. In this 24-week open-label Phase 3 study, children (N=66) weighing <30 kg received 50% of the ELX/TEZ/IVA adult daily dose (ELX 100 mg once daily, TEZ 50 mg once daily, and IVA 75 mg every 12 hours) while children weighing ≥30 kg received the full adult daily dose (ELX 200 mg once daily, TEZ 100 mg once daily, and IVA 150 mg every 12 hours). The primary endpoint was safety and tolerability. The safety and pharmacokinetic profiles of ELX/TEZ/IVA were generally consistent with those observed in older patients. The most common reported adverse events (AEs) included cough, headache, and pyrexia; in most of the children who had AEs, these were mild or moderate in severity. Through Week 24, ELX/TEZ/IVA treatment improved the ppFEV1 (10.2 percentage points; 95% confidence interval [CI], 7.9 to 12.6), Cystic Fibrosis Questionnaire-Revised respiratory domain score (7.0 points; 95% CI, 4.7 to 9.2), lung clearance index2.5 (-1.71 units; 95% CI, -2.11 to -1.30), and sweat chloride (-60.9 mmol/L; 95% CI, -63.7 to -58.2); body mass index-for-age z-score increased over the 24-week treatment period compared to the pre-treatment baseline. Our results show ELX/TEZ/IVA is safe and efficacious in children 6 through 11 years of age with at least one F508del-CFTR allele, supporting its use in this patient population. Clinical trial registration available at, ID: NCT03691779.

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