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@article{Zemanick:2021:10.1164/rccm.202102-0509OC,
author = {Zemanick, ET and Taylor-Cousar, JL and Davies, J and Gibson, RL and Mall, MA and McKone, EF and McNally, P and Ramsey, BW and Rayment, JH and Rowe, SM and Tullis, E and Ahluwalia, N and Chu, C and Ho, T and Moskowitz, SM and Noel, S and Tian, S and Waltz, D and Weinstock, TG and Xuan, F and Wainwright, CE and McColley, SA and VX18-445-106, Study Group},
doi = {10.1164/rccm.202102-0509OC},
journal = {American Journal of Respiratory and Critical Care Medicine},
pages = {1522--1532},
title = {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.},
url = {http://dx.doi.org/10.1164/rccm.202102-0509OC},
volume = {203},
year = {2021}
}

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TY  - JOUR
AB  - RATIONALE: 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. OBJECTIVES: 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. METHODS: 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). MEASUREMENTS AND MAIN RESULTS: 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. CONCLUSIONS: 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 www.clinicaltrials.gov, ID: NCT03691779 This article is open access and distributed under the terms of the Creative Commons Attribution Non-Commercial No D
AU  - Zemanick,ET
AU  - Taylor-Cousar,JL
AU  - Davies,J
AU  - Gibson,RL
AU  - Mall,MA
AU  - McKone,EF
AU  - McNally,P
AU  - Ramsey,BW
AU  - Rayment,JH
AU  - Rowe,SM
AU  - Tullis,E
AU  - Ahluwalia,N
AU  - Chu,C
AU  - Ho,T
AU  - Moskowitz,SM
AU  - Noel,S
AU  - Tian,S
AU  - Waltz,D
AU  - Weinstock,TG
AU  - Xuan,F
AU  - Wainwright,CE
AU  - McColley,SA
AU  - VX18-445-106,Study Group
DO  - 10.1164/rccm.202102-0509OC
EP  - 1532
PY  - 2021///
SN  - 1073-449X
SP  - 1522
TI  - 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.
T2  - American Journal of Respiratory and Critical Care Medicine
UR  - http://dx.doi.org/10.1164/rccm.202102-0509OC
UR  - https://www.ncbi.nlm.nih.gov/pubmed/33734030
UR  - https://www.atsjournals.org/doi/10.1164/rccm.202102-0509OC
UR  - http://hdl.handle.net/10044/1/87673
VL  - 203
ER  -

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