Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation
Summary
The purpose of this study is to evaluate of the safety, efficacy, pharmacokinetics (PK) and pharmacodynamic (PD) effects of lumacaftor (VX-809) alone and when coadministered with ivacaftor (VX-770) in participants with cystic fibrosis, homozygous or heterozygous for the F508del-CFTR mutation.
Timeline
- Start
- 2010-10
- Primary completion
- 2014-04
- Completion
- 2014-04
Outcome
Sources disagree · no verdict is set; each source's reading is shown below
registry p <0.001; LS Mean difference -9.676 (95% CI -14.801 to -4.551); ANCOVA reads as mixed primary results NCT01225211 ↗
registry p = 0.680; LS Mean difference -1.306 (95% CI -7.565 to 4.953); ANCOVA reads as mixed primary results NCT01225211 ↗
registry p = 0.5978; LS Mean difference 0.60 (95% CI -1.66 to 2.86); Mixed Model Repeated Measure (MMRM) reads as mixed primary results NCT01225211 ↗
release “The first part of the study met its two primary endpoints: (1) safety and tolerability of the combination regimen” reads as met primary endpoint vrtx.com ↗
release “met its two primary endpoints: (1) safety and tolerability of the combination regimen and (2) the effect of the combination of VX-770 and VX-809” reads as met primary endpoint vrtx.com ↗
Publications
- Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.
- Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
- Rowe SM, McColley SA, Rietschel E, Li X, Bell SC, Konstan MW, Marigowda G, Waltz D, Boyle MP; VX09-809-102 Study Group. Lumacaftor/Ivacaftor Treatment of Patients with Cystic Fibrosis Heterozygous for F508del-CFTR. Ann Am Thorac Soc. 2017 Feb;14(2):213-219. doi: 10.1513/AnnalsATS.201609-689OC.
- Boyle MP, Bell SC, Konstan MW, McColley SA, Rowe SM, Rietschel E, Huang X, Waltz D, Patel NR, Rodman D; VX09-809-102 study group. A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial. Lancet Respir Med. 2014 Jul;2(7):527-38. doi: 10.1016/S2213-2600(14)70132-8. Epub 2014 Jun 24.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Ivacaftor | Other / unclassified | 150 mg | — |
| Subject | Ivacaftor | Other / unclassified | 250 mg | — |
| Subject | Lumacaftor | Small molecule | 200 mg | — |
| Subject | Lumacaftor | Small molecule | 400 mg | — |
| Subject | Lumacaftor | Small molecule | 600 mg | — |