VX17-445-102 Efficacy and Safety of VX-445/TEZ/IVA in CF Het-min
Details
Therapeutic approach
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
A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
Study details
A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
Study results
This study evaluated the safety and efficacy of ELX/TEZ/IVA (also known as Kaftrio) in patients aged 12 years and older with one F508del mutation and one minimal function mutation.
403 people took part in the study and were split into two groups – one group received 24 weeks of Kaftrio treatment and the other group received no treatment (placebo). The study measured changes in lung function, sweat chloride, health-related quality of life, and other health-related outcomes.
People taking Kaftrio had improved lung function, fewer pulmonary exacerbations, decreased sweat chloride, and an improved health-related quality of life. Most adverse events experienced by patients in the treatment group were mild or moderate.
Kaftrio treatment was generally safe and effective for people with cystic fibrosis ages 12+ with Phe508del-minimal function genotypes for whom previous CFTR modulator treatments were not effective. These results support Kaftrio as a treatment for people with these gene variants.
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 3
Recruitment target
The number of participants who need to be recruited for the trial in the UK
360
CF sponsor
Vertex Pharmaceuticals Incorporated
CF sponsor type
Commercial
Who can take part?
Age range
12 years and older
Including people
Age 12 Years and older
Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
FEV1 above or equal to 40% and below or equal to 90%
