VX17-445-103 Efficacy and Safety of VX-445/TEZ/IVA in CF Homozygous
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 Homozygous for the F508del Mutation (F/F)
Study details
To evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and VX-561 (deuterated ivacaftor, also known as CTP-656) in subjects with cystic fibrosis (CF)who are homozygous for the F508del mutation (F/F)
Study results
This study evaluated the safety and efficacy of ELX/TEZ/IVA (also known as Kaftrio) in people with CF aged 12 years and older with two copies of the F508del mutation.
113 people took part in the study. There was a 4-week ‘run-in’ period where all participants were treated with tezacaftor plus ivacaftor. Following the run-in period, participants were split into two groups – one group received 4 weeks of Kaftrio treatment and the other group continued with 4 more weeks of tezacaftor plus ivacaftor treatment alone.
People receiving Kaftrio showed improved lung function, decreased sweat chloride, and better health-related quality of life compared to the other group. Kaftrio was well-tolerated; most adverse events were mild or moderate.
These results suggest Kaftrio could be a more effective treatment than tezacaftor plus ivacaftor alone for people with cystic fibrosis with two copies of the F508del mutation.
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
100
CF sponsor
Vertex Pharmaceuticals Incorporated
CF sponsor type
Commercial
Who can take part?
Age range
12 years and older
Excluding people
Clinically significant cirrhosis with or without portal hypertension.
