drugset / Trial / NCT03625466
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
RandomizedParallel-groupQuadruple-blindTreatment
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
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
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Ivacaftor | Other / unclassified | 125 mg | Oral |
| Subject | Ivacaftor | Other / unclassified | 188 mg | Oral |
| Subject | Ivacaftor | Other / unclassified | 250 mg | Oral |
| Subject | Lumacaftor | Small molecule | 100 mg | Oral |
| Subject | Lumacaftor | Small molecule | 150 mg | Oral |
| Subject | Lumacaftor | Small molecule | 200 mg | Oral |