A Study to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del

Summary

This study will explore the impact of lumacaftor/ivacaftor (LUM/IVA) on disease progression in subjects aged 2 through 5 years with cystic fibrosis (CF), homozygous for F508del (F/F).

Timeline

Start

2018-08-10

Primary completion

2020-10-09

Completion

2021-10-07

Outcome

Missed primary endpoint

registry analysis (superiority test); Part 1: Placebo vs Part 1: LUM/IVA; Mean Difference -1.5 (95% CI -5.5 to 2.6) NCT03625466 ↗

Publications

• Stahl M, Roehmel J, Eichinger M, Doellinger F, Naehrlich L, Kopp MV, Dittrich AM, Sommerburg O, Ray P, Maniktala A, Xu T, Conner S, Joshi A, Mascia M, Wielputz MO, Mall MA. Long-Term Impact of Lumacaftor/Ivacaftor Treatment on Cystic Fibrosis Disease Progression in Children 2-5 Years of Age Homozygous for F508del-CFTR: A Phase 2, Open-Label Clinical Trial. Ann Am Thorac Soc. 2024 Nov;21(11):1550-1559. doi: 10.1513/AnnalsATS.202402-201OC.
• Stahl M, Roehmel J, Eichinger M, Doellinger F, Naehrlich L, Kopp MV, Dittrich AM, Lee C, Sommerburg O, Tian S, Xu T, Wu P, Joshi A, Ray P, Duncan ME, Wielputz MO, Mall MA. Effects of Lumacaftor/Ivacaftor on Cystic Fibrosis Disease Progression in Children 2 through 5 Years of Age Homozygous for F508del-CFTR: A Phase 2 Placebo-controlled Clinical Trial. Ann Am Thorac Soc. 2023 Aug;20(8):1144-1155. doi: 10.1513/AnnalsATS.202208-684OC.

Drugs

Indications