Phase 3 Open-Label Clinical Trial of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged 2-5 Years with Cystic Fibrosis and at Least One F508del Allele

Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) has been shown to be safe and effective in people with cystic fibrosis (CF) aged ⩾6 years with at least one allele but has not been studied in younger children. To evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of ELX/TEZ/IVA in c...

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Published in:American journal of respiratory and critical care medicine 2023-07, Vol.208 (1), p.59-67
Main Authors: Goralski, Jennifer L, Hoppe, Jordana E, Mall, Marcus A, McColley, Susanna A, McKone, Edward, Ramsey, Bonnie, Rayment, Jonathan H, Robinson, Phil, Stehling, Florian, Taylor-Cousar, Jennifer L, Tullis, Elizabeth, Ahluwalia, Neil, Chin, Anna, Chu, Chenghao, Lu, Mengdi, Niu, Tao, Weinstock, Tanya, Ratjen, Felix, Rosenfeld, Margaret
Format: Article
Language:English
Subjects:
Aged
Alleles
Aminophenols
Benzodioxoles
Child
Children & youth
Chloride Channel Agonists - therapeutic use
Chlorides
Clinical trials
Cystic fibrosis
Cystic Fibrosis - drug therapy
Cystic Fibrosis - genetics
Cystic Fibrosis Transmembrane Conductance Regulator - genetics
Cystic Fibrosis Transmembrane Conductance Regulator - therapeutic use
Humans
Mutation
Pharmacokinetics
Pulmonary fibrosis
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Summary:Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) has been shown to be safe and effective in people with cystic fibrosis (CF) aged ⩾6 years with at least one allele but has not been studied in younger children. To evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of ELX/TEZ/IVA in children with CF aged 2-5 years. In this phase 3, open-label, two-part study (parts A and B), children weighing
ISSN:1073-449X
1535-4970
DOI:10.1164/rccm.202301-0084OC