A study to evaluate the safety and effectiveness of a new triple combination CFTR modulator therapy in children aged 1 to 11 (VX21-121-105)

Full title

A phase 3 study evaluating the safety, tolerability and effectiveness of VX-121/TEZ/D-IVA triple combination therapy in children aged 1 to 11

Study details

This study aims to evaluate whether a new triple combination CFTR modulator treatment (VX-121/TEZ/D-IVA) is safe and effective in children with cystic fibrosis aged between 1 and 11 years.

CFTR modulators help the faulty CFTR protein to function properly.

The treatment is made up of three medications: VX-121, tezacaftor and deutivacaftor. Deutivacaftor is a slightly modified version of ivacaftor that is potentially more stable in the body. This means deutivacaftor can be taken once a day instead of twice a day.

All participants will be given an oral tablet of the treatment to take once a day. The researchers will monitor participants’ overall health. They will measure the concentration of the drug in the blood by taking blood samples, and they may take other measurements such as sweat chloride levels and weight. Participation will last for around 6 months.

Children with at least one CF mutation which is responsive to triple combination CFTR modulators (e.g. Kaftrio) may be eligible to take part in this study (see the inclusion and exclusion criteria list for more details).

Please note: This study has completed recruitment for the 6 to 11 years age group and is not currently open for the younger age groups. Please stay tuned for further information. 

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