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A study of the combined long-term effects of three CFTR modulator medicines in people with cystic fibrosis aged 12 and older (VX18 - 445 - 110)

Details

Therapeutic category
the type of treatment or therapy being studied. A therapy could range from a medication addressing a particular characteristic of CF to a device or activity e.g. exercise

Restore CFTR Function

Trial status

Closed

Phase
the different stages involved in the development of a new medication. Phase I focuses on initial safety in people. Phase 2 evaluates safety, correct dose and early signs of whether the medication works. Phase 3 is the stage before medication licensing and looks at safety and medication effectiveness. Phase 4 evaluates longer term use of a medication after it has been licensed for use

Phase III

Full title

A Phase 3 study of the long-term safety and efficacy of a triple combination therapy of VX-445, tezacaftor and ivacaftor in people with cystic fibrosis aged 12 and older who have one copy of the F508del mutation and one copy of a gating or residual function mutation

In order to take part in this study you will already be taking part in the VX18-445-104 study. VX-445 is a medicine being studied for how well it works and how safe it is in people with cystic fibrosis (CF). Your participation in this study will help us learn more about how well VX-445 works in combination with tezacaftor and ivacaftor in people with CF with one copy of a CF gene with a mutation termed F508del and the other copy of a CF gene with a mutation termed gating or residual function. All three of the medicines are CFTR modulators which means that they help the faulty CFTR protein to function properly. Tezacaftor is already approved for use and is what is known as a corrector. Ivacaftor is also already approved for use and is what is known as a potentiator. VX-445 is a corrector. Everyone in the study will get the study medications. If you meet the rules for being in the study, you will take VX-445, tezacaftor and ivacaftor in the morning (in 2 pills), and ivacaftor in the evening (1 pill). You will be asked to come into clinic around 13 times over up to 100 weeks. During visits, we will measure how the drug is affecting your lung function and other measures of health with bloods and other samples, lung function, vital signs and other investigations. You will also receive 14 telephone consultations, each around half an hour, during this period. You do not have to come to clinics for these.

Trial Reference Number

124415

Trial type

Medication

Intervention
the name of the treatment or therapy being researched

CFTR Modulators

Last edited date

12 June 2020

CF sponsor

Vertex Pharmaceuticals Incorporated

CF sponsor type

Commercial

Who can take part?

Top inclusion criteria
  • 12 Years and older
  • Currently participating in study VX18-445-104
Top exclusion criteria
  • History of intolerance in study VX18-445-104
  • Current participation in an investigational drug trial (other than study VX18-445-104)