Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor

Details

Therapeutic category
Restore CFTR Function
Trial status
Completed Participating Centres
Phase
Phase III

Full title

A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Longterm Treatment With Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Years and Older With Cystic Fibrosis, Homozygous for the F508delCFTR Mutation

This study will look at whether two drugs (called lumacaftor and ivacaftor) can help treat cystic fibrosis when taken together over a long period of time. It will also look at whether they are safe over this period.

You can only take part in this study if you took part in a study called VX14-809-109, and if you meet certain criteria. These criteria will be checked by the study doctor and nurse at a screening visit. If you meet these criteria you will be treated with lumacaftor and ivacaftor together for approximately 96 weeks. A follow up visit will be held 28 days after the last dose of lumacaftor and ivacaftor. You will need to attend the clinic for 15 visits over the study duration and will undergo a range of tests.

Participants who stopped taking the study drugs early in the VX14-809-109 study will be invited to take part in an observational study. They will not receive the study drug but will receive regular phone calls.

Trial Reference Number
50600
Trial type
Medication
Length of participation
96 weeks
Intervention
CFTR Modulators (Ivacaftor, Lumacaftor)
Recruitment target
15
Last edited date
01/07/2019
CF sponsor
Vertex Pharmaceuticals Incorporated
CF sponsor type
Commercial

Who can take part?

Age
6+
Top inclusion criteria
  • Homozygous for the F508del-CFTR mutation
  • Weight >= 15Kg

Your donation will make a difference:

Select amount