Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 Through 11 Years of Age with Cystic Fibrosis Heterozygous for F508del and a Minimal Function Mutation: A Phase 3b, Randomized, Placebo-controlled Study

Author:

Mall Marcus A.123ORCID,Brugha Rossa4,Gartner Silvia5,Legg Julian6,Moeller Alexander7,Mondejar-Lopez Pedro8,Prais Dario910,Pressler Tacjana11,Ratjen Felix12,Reix Philippe13,Robinson Paul D.14,Selvadurai Hiran14,Stehling Florian15,Ahluwalia Neil16,Arteaga-Solis Emilio16,Bruinsma Bote G.16,Jennings Mark16,Moskowitz Samuel M.16,Noel Sabrina16,Tian Simon16,Weinstock Tanya G.16,Wu Pan16,Wainwright Claire E.17ORCID,Davies Jane C.181920

Affiliation:

1. Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine, Charité – Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin and

2. Berlin Institute of Health at Charité – Universitätsmedizin Berlin, Berlin, Germany;

3. German Center for Lung Research, Associated Partner, Berlin, Germany;

4. Great Ormond Street Hospital for Children, London, United Kingdom;

5. Hospital Universitari Vall d’Hebron, Barcelona, Spain;

6. Southampton Children's Hospital, Hampshire, United Kingdom;

7. University Children’s Hospital, Zurich, Switzerland;

8. Hospital Clinico Universitario Virgen de la Arrixaca, Murcia, Spain;

9. Schneider Children’s Medical Center of Israel, Petah Tikva, Israel;

10. Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel;

11. Copenhagen University Hospital, Rigshospitalet, Denmark;

12. The Hospital for Sick Children, Toronto, Ontario, Canada;

13. Hôpital Femme Mère-Enfant, Hospices Civils de Lyon, Bron, France;

14. The Children’s Hospital at Westmead, Sydney Children’s Hospital Network, Sydney, Australia;

15. Universitätsklinikum Essen, Klinik für Kinderheilkunde III, Essen, Germany;

16. Vertex Pharmaceuticals Incorporated, Boston, Massachussetts;

17. Queensland Children’s Hospital, University of Queensland, Queensland, Australia;

18. National Heart and Lung Institute, Imperial College London, London, United Kingdom;

19. Royal Brompton and Harefield Hospitals, part of Guy’s and St Thomas’ NHS Trust, London, United Kingdom; and

20. European Cystic Fibrosis Society Lung Clearance Index Core Facility, London, United Kingdom

Funder

Vertex Pharmaceuticals

Publisher

American Thoracic Society

Subject

Critical Care and Intensive Care Medicine,Pulmonary and Respiratory Medicine

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