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A study of three CFTR modulator medicines for how well they work together and their safety in people with cystic fibrosis aged 6 to 11 with one F508del mutation and one minimal function mutation (VX19-445-116)

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

Open to recruitment

Participating Centres
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 IIIb

Full title

A Phase 3b randomised, placebo-controlled study of the efficacy and safety of a triple combination therapy of elexacaftor (VX-445), tezacaftor and ivacaftor in people with cystic fibrosis aged 6 to 11 with one F508del mutation and one minimal function mutation

In order to take part in this study you will have one F508del mutation and one minimal function mutation, and be aged 6 to 11. Elexacaftor (VX-445) is a medicine being studied for how well it works and how safe it is in people with cystic fibrosis. Your participation in this study will help us learn more about the safety of taking elexacaftor in combination with tezacaftor and ivacaftor. 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. Elexacaftor is a corrector. If you meet the rules for being in the study, you will take either: a combination of elexacaftor, tezacaftor and ivacaftor in the morning (in 2 tablets), and ivacaftor in the evening (1 tablet);or the same number of placebo tablets in the morning and evening. You will not know whether you are taking the study medications or a placebo. You will be asked to come into clinic around 9 times over up to 32 weeks. During visits, we will look for any changes in your health with bloods and other samples, vital signs and other investigations. You will also receive 3 telephone consultations, each around half an hour, during this period. You do not have to come to clinics for these.

Trial Reference Number

128538

Intervention
the name of the treatment or therapy being researched

CFTR Modulators

Last edited date

22 June 2020

CF sponsor

Vertex Pharmaceuticals Incorporated

CF sponsor type

Commercial

Who can take part?

Top inclusion criteria
  • 6 Years to 11 Years
  • Heterozygous for the F508del mutation
  • FEV1 value greater than equal to 70%
Top exclusion criteria
  • Cirrhosis with or without portal hypertension

CF centres running this trial

Recruiting

Alder Hey Children's Hospital

Address

Alder Hey Hospital Eaton Road West Derby Liverpool Merseyside L12 2AP

Recruitment starts

July 2020

Recruitment ends

November 2020

Contact

Thursfield, Rebecca

Get in touch

Recruiting

Bristol Royal Hospital for Children

Address

Upper Maudlin Street Bristol Avon BS2 8BJ

Recruitment starts

September 2020

Recruitment ends

December 2020

Contact

Hilliard, Tom

Get in touch

Closed

Royal Brompton Hospital

Address

Sydney Street London SW3 6NP

Recruitment starts

August 2020

Recruitment ends

October 2020

Contact

Davies, Jane C

Get in touch