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@article{Wainwright:2023:10.1164/rccm.202301-0021OC,
author = {Wainwright, C and McColley, SA and McNally, P and Powers, M and Ratjen, F and Rayment, JH and Retsch-Bogart, G and Roesch, E and Ahluwalia, N and Chin, A and Chu, C and Lu, M and Menon, P and Waltz, D and Weinstock, T and Zelazoski, L and Davies, JC},
doi = {10.1164/rccm.202301-0021OC},
journal = {American Journal of Respiratory and Critical Care Medicine},
pages = {68--78},
title = {Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in children aged 6 years with cystic fibrosis and at least one F508del allele: a phase 3, open-label clinical trial.},
url = {http://dx.doi.org/10.1164/rccm.202301-0021OC},
volume = {208},
year = {2023}
}

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TY  - JOUR
AB  - Rationale: A 24-week, phase 3, open-label study showed elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was safe and efficacious in children aged 6-11 years with cystic fibrosis (CF) and one or more F508del-CFTR alleles. Objectives: To assess long-term safety and efficacy of ELX/TEZ/IVA in children who completed the pivotal 24-week phase 3 trial. Methods: In this phase 3, two-part (part A and part B), open-label extension study, children aged 6 years with CF heterozygous for F508del and a minimal function CFTR mutation (F/MF genotypes) or homozygous for F508del (F/F genotype) who completed the 24-week parent study received ELX/TEZ/IVA based on weight. Children weighing <30 kg received ELX 100 mg once daily/TEZ 50 mg once daily/IVA 75 mg every 12 hours, whereas children weighing 30 kg received ELX 200 mg once daily/TEZ 100 mg once daily/IVA 150 mg every 12 hours (adult dose). The 96-week analysis of part A of this extension study is reported here. Measurements and Main Results: Sixty-four children (F/MF genotypes, n = 36; F/F genotype, n = 28) were enrolled and received one or more doses of ELX/TEZ/IVA. Mean (SD) period of exposure to ELX/TEZ/IVA was 93.9 (11.1) weeks. The primary endpoint was safety and tolerability. Adverse events and serious adverse events were consistent with common manifestations of CF disease. Overall, exposure-adjusted rates of adverse events and serious adverse events (407.74 and 4.72 events per 100 patient-years) were lower than in the parent study (987.04 and 8.68 events per 100 patient-years). One child (1.6%) had an adverse event of aggression that was moderate in severity and resolved after study drug discontinuation. From parent study baseline at Week 96 of this extension study, the mean percent predicted FEV1 increased (11.2 [95% confidence interval (CI), 8.3 to 14.2] percentage points), sweat chloride
AU  - Wainwright,C
AU  - McColley,SA
AU  - McNally,P
AU  - Powers,M
AU  - Ratjen,F
AU  - Rayment,JH
AU  - Retsch-Bogart,G
AU  - Roesch,E
AU  - Ahluwalia,N
AU  - Chin,A
AU  - Chu,C
AU  - Lu,M
AU  - Menon,P
AU  - Waltz,D
AU  - Weinstock,T
AU  - Zelazoski,L
AU  - Davies,JC
DO  - 10.1164/rccm.202301-0021OC
EP  - 78
PY  - 2023///
SN  - 1073-449X
SP  - 68
TI  - Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in children aged 6 years with cystic fibrosis and at least one F508del allele: a phase 3, open-label clinical trial.
T2  - American Journal of Respiratory and Critical Care Medicine
UR  - http://dx.doi.org/10.1164/rccm.202301-0021OC
UR  - https://www.ncbi.nlm.nih.gov/pubmed/37154609
UR  - https://www.atsjournals.org/doi/10.1164/rccm.202301-0021OC
UR  - http://hdl.handle.net/10044/1/106044
VL  - 208
ER  -

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