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A study of the combined effects of three CFTR modulator medicines in children with cystic fibrosis aged 6 to 11 (VX18-445-106)

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 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 III

Full title

A Phase 3 study of the efficacy, safety and the body’s effects on a triple combination therapy of VX-445, tezacaftor and ivacaftor in children with cystic fibrosis aged 6 to 11

VX-445 is an investigational medicine being studied to see how well it works and how safe it is in children with cystic fibrosis aged 6 to 11 years, when taken in combination with tezacaftor and ivacaftor. All three medicines are CFTR modulators which means that they help the faulty CFTR protein found in cystic fibrosis to work 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. Trial participants will be asked to take VX-445, tezacaftor and ivacaftor in the morning (2 tablets), and ivacaftor in the evening (1 tablet). Everyone in the study will receive all three of the study medications, no placebo is involved. This study is divided into two parts. Part A will recruit 12 people who will be asked to come into clinic around 6 times over 10 weeks. Part B will recruit 56 people who will be asked to come into clinic around 11 times over 32 weeks. During visits, the effect of the treatment on your CF will be measured with sample collection, lung function, questionnaires, and other medical checks.

Trial Reference Number

126329

Trial type

Medication

Intervention
the name of the treatment or therapy being researched

CFTR Modulators

Last edited date

15 November 2019

CF sponsor

Vertex Pharmaceuticals Incorporated

CF sponsor type

Commercial

Who can take part?

Top inclusion criteria
  • 6 Years to 11 Years
  • Homozygous or heterozygous for F508del mutation
Top exclusion criteria
  • Lung infection

CF centres running this trial

Closed

Birmingham Children's Hospital

Address

Steelhouse Lane Birmingham West Midlands B4 6NH

Recruitment starts

November 2019

Recruitment ends

January 2020

Contact

Desai, Maya

Get in touch

Closed

Royal Brompton Hospital

Address

Sydney Street London SW3 6NP

Contact

Davies, Jane C

Get in touch