drugset / Trial / NCT04353817
A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis and F/MF Genotypes
RandomizedParallel-groupQuadruple-blindTreatment
Summary
This study will evaluate the efficacy and safety of elexacaftor (ELX) / tezacaftor (TEZ) / ivacaftor (IVA) triple combination (TC) in subjects 6 through 11 years of age with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function (MF) mutation (F/MF genotypes).
Timeline
- Start
- 2020-06-19
- Primary completion
- 2021-05-17
- Completion
- 2021-05-17
Outcome
Met primary endpoint
registry analysis (superiority test); Placebo vs ELX/TEZ/IVA; p <0.0001; Least Squares (LS) Mean Difference -2.26 (95% CI -2.71 to -1.81) NCT04353817 ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | ELX/TEZ/IVA | Unknown | — | Oral |
| Subject | IVA | Unknown | 75 mg | Oral |
| Subject | IVA | Unknown | 150 mg | Oral |