Safety and efficacy of vanzacaftor–tezacaftor–deutivacaftor in children with cystic fibrosis aged 2–5 years (TIMBERLINE Trial VX21-121-105): a phase 3, open-label study

Jonathan H Rayment, Gwyneth Davies, Jonathan M Gaffin, Jordana E Hoppe, Ajay S Kasi, Marcus A Mall, Edward F McKone, Bonnie Ramsey, Jennifer L Taylor, Elizabeth Tullis, Emily McDougall, Robin Pottackal, Rachel Zahigian, Katie Krone, Mark Jennings, James F Chmiel, Gary McPhail, Erica Roesch, Daniel Weiner, Carlos Milla, Jessica Pittman, Rachel Linnemann, Hugo Escobar, Fadel Ruiz, Thomas Lahiri, Brooke Moore, Alexander Moeller, Anna-Maria Dittrich, Felix Ratjen, Pornchai Tirakitsoontorn, Maria Dowell, Lena Thia, Catherine Byrnes, Florian Stehling, Isabelle Sermet-Gaudelus, Philippe Reix, Philip Robinson, Claire Wainwright, David Graeme Hancock, Andrew Tai, Isabelle De Monestrol

Open source

DOI
10.1016/s2213-2600(26)00257-2
Published
2026-09
Container
The Lancet Respiratory Medicine
Publisher
Elsevier BV
Open access
unknown

Credibility signals

uncertain Score 64/100 under policy 1.0.0. This is a metadata assessment, not a judgment of the paper's conclusions.

Show all credibility signals

Cite this work

BibTeX

@article{allodium:10.1016/s2213-2600-26-00257-2,
  title = {Safety and efficacy of vanzacaftor–tezacaftor–deutivacaftor in children with cystic fibrosis aged 2–5 years (TIMBERLINE Trial VX21-121-105): a phase 3, open-label study},
  author = {Jonathan H Rayment and Gwyneth Davies and Jonathan M Gaffin and Jordana E Hoppe and Ajay S Kasi and Marcus A Mall and Edward F McKone and Bonnie Ramsey and Jennifer L Taylor and Elizabeth Tullis and Emily McDougall and Robin Pottackal and Rachel Zahigian and Katie Krone and Mark Jennings and James F Chmiel and Gary McPhail and Erica Roesch and Daniel Weiner and Carlos Milla and Jessica Pittman and Rachel Linnemann and Hugo Escobar and Fadel Ruiz and Thomas Lahiri and Brooke Moore and Alexander Moeller and Anna-Maria Dittrich and Felix Ratjen and Pornchai Tirakitsoontorn and Maria Dowell and Lena Thia and Catherine Byrnes and Florian Stehling and Isabelle Sermet-Gaudelus and Philippe Reix and Philip Robinson and Claire Wainwright and David Graeme Hancock and Andrew Tai and Isabelle De Monestrol},
  year = {2026},
  journal = {The Lancet Respiratory Medicine},
  doi = {10.1016/s2213-2600(26)00257-2},
  url = {https://doi.org/10.1016/s2213-2600(26)00257-2}
}

RIS

TY  - JOUR
TI  - Safety and efficacy of vanzacaftor–tezacaftor–deutivacaftor in children with cystic fibrosis aged 2–5 years (TIMBERLINE Trial VX21-121-105): a phase 3, open-label study
AU  - Jonathan H Rayment
AU  - Gwyneth Davies
AU  - Jonathan M Gaffin
AU  - Jordana E Hoppe
AU  - Ajay S Kasi
AU  - Marcus A Mall
AU  - Edward F McKone
AU  - Bonnie Ramsey
AU  - Jennifer L Taylor
AU  - Elizabeth Tullis
AU  - Emily McDougall
AU  - Robin Pottackal
AU  - Rachel Zahigian
AU  - Katie Krone
AU  - Mark Jennings
AU  - James F Chmiel
AU  - Gary McPhail
AU  - Erica Roesch
AU  - Daniel Weiner
AU  - Carlos Milla
AU  - Jessica Pittman
AU  - Rachel Linnemann
AU  - Hugo Escobar
AU  - Fadel Ruiz
AU  - Thomas Lahiri
AU  - Brooke Moore
AU  - Alexander Moeller
AU  - Anna-Maria Dittrich
AU  - Felix Ratjen
AU  - Pornchai Tirakitsoontorn
AU  - Maria Dowell
AU  - Lena Thia
AU  - Catherine Byrnes
AU  - Florian Stehling
AU  - Isabelle Sermet-Gaudelus
AU  - Philippe Reix
AU  - Philip Robinson
AU  - Claire Wainwright
AU  - David Graeme Hancock
AU  - Andrew Tai
AU  - Isabelle De Monestrol
PY  - 2026
JO  - The Lancet Respiratory Medicine
DO  - 10.1016/s2213-2600(26)00257-2
UR  - https://doi.org/10.1016/s2213-2600(26)00257-2
ER  - 

APA

Rayment, J. H., Davies, G., Gaffin, J. M., Hoppe, J. E., Kasi, A. S., Mall, M. A., McKone, E. F., Ramsey, B., Taylor, J. L., Tullis, E., McDougall, E., Pottackal, R., Zahigian, R., Krone, K., Jennings, M., Chmiel, J. F., McPhail, G., Roesch, E., Weiner, D., Milla, C., Pittman, J., Linnemann, R., Escobar, H., Ruiz, F., Lahiri, T., Moore, B., Moeller, A., Dittrich, A., Ratjen, F., Tirakitsoontorn, P., Dowell, M., Thia, L., Byrnes, C., Stehling, F., Sermet-Gaudelus, I., Reix, P., Robinson, P., Wainwright, C., Hancock, D. G., Tai, A., & Monestrol, I. D. (2026). Safety and efficacy of vanzacaftor–tezacaftor–deutivacaftor in children with cystic fibrosis aged 2–5 years (TIMBERLINE Trial VX21-121-105): a phase 3, open-label study. The Lancet Respiratory Medicine. https://doi.org/10.1016/s2213-2600(26)00257-2

Source records