Study of VX-661 Alone and in Combination With Ivacaftor in Subjects Homozygous or Heterozygous to the F508del-Cystic Fibrosis Transmembrane Conductance Regulator(CFTR) Mutation
Summary
The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) effects of VX-661 alone and when coadministered with ivacaftor in participants with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del-CFTR mutation.
Timeline
- Start
- 2012-02
- Primary completion
- 2014-03
- Completion
- 2014-03
Outcome
Mixed primary results
registry analysis (superiority test); Group 2b: VX-661 10 mg qd/Ivacaftor 150 mg q12h vs Group 1-5b Combined Placebo; p = 0.0348; LS Mean Difference -4.2 (95% CI -8.1 to -0.31); MMRM NCT01531673 ↗
registry analysis (superiority test); Group 5b: VX-661 150 mg qd/Ivacaftor 150 mg q12h vs Group 1-5b Combined Placebo; p = 0.3745; LS Mean Difference -1.77 (95% CI -5.71 to 2.17); MMRM NCT01531673 ↗
registry analysis (superiority test); Group 6d: VX-661 50 mg q12h/Ivacaftor 150 mg q12h vs Group 4 and 6 Combined: Placebo; p = 0.0357; LS Mean Difference -6.7 (95% CI -12.94 to -0.46); MMRM NCT01531673 ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Ivacaftor | Other / unclassified | 50 mg | Oral |
| Subject | Ivacaftor | Other / unclassified | 150 mg | Oral |
| Subject | tezacaftor | Other / unclassified | 10 mg | Oral |
| Subject | tezacaftor | Other / unclassified | 30 mg | Oral |
| Subject | tezacaftor | Other / unclassified | 50 mg | Oral |
| Subject | tezacaftor | Other / unclassified | 100 mg | Oral |
| Subject | tezacaftor | Other / unclassified | 150 mg | Oral |