Rationale: Elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) was shown to be safe and efficacious in children 6 through 11 years of age with cystic fibrosis (CF) and at least one F508del allele in a 24-week phase 3 study. Children completing this study could enroll into a 192-week extension study. Objectives: To evaluate the long-term safety and efficacy of ELX/TEZ/IVA in children
- Wainwright, C.
- McColley, S. A.
- McNally, P.
- Powers, M.
- Ratjen, F.
- Rayment, J. H.
- Retsch-Bogart, G.
- Roesch, E.
- Ramsey, B.
- McKone, E. F.
- Tullis, E.
- Mall, M. A.
- Taylor-Cousar, J. L.
- Waltz, D.
- Ahluwalia, N.
- Chu, C.
- Scirica, C. V.
- Davies, J. C.
Keywords
- Humans
- Child
- *Cystic Fibrosis/drug therapy/genetics
- *Aminophenols/therapeutic use/adverse effects/administration & dosage
- *Quinolones/therapeutic use/adverse effects/administration & dosage
- Male
- Female
- *Benzodioxoles/therapeutic use/adverse effects/administration & dosage
- Drug Combinations
- *Indoles/therapeutic use/adverse effects/administration & dosage
- Treatment Outcome
- Cystic Fibrosis Transmembrane Conductance Regulator/genetics
- *Pyrazoles/therapeutic use/adverse effects/administration & dosage
- Alleles
- *Quinoxalines/therapeutic use/adverse effects/administration & dosage
- *Phenylpropionates/therapeutic use/adverse effects/administration & dosage
- Chloride Channel Agonists/therapeutic use
- Pyrrolidines/therapeutic use
- *Pyridines/therapeutic use/adverse effects
- Quinolines
- children
- cystic fibrosis transmembrane conductance regulator modulator
- elexacaftor/tezacaftor/ivacaftor
- long-term extension study
