Trials Tracker

A long-term extension study of the combined effects of three CFTR modulator medicines in people with cystic fibrosis aged 6 and older (VX19 445 107)

VX-445 is a medicine being studied for how well it works and how safe it is in people with cystic fibrosis. This study is an extension of existing study (VX18-445-106) to look at the long-term efficacy and safety of VX-445 in combination with tezacaftor and ivacaftor. All three are CFTR modulators which means that they help the faulty CFTR protein 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. Participants from the previous study who chose to take part in this extension will take VX-445, tezacaftor and ivacaftor in the morning (in 2 tablets), and ivacaftor in the evening (1 tablet). The study will involve up to 11 clinic visits and 15 telephone consultations over 100 weeks (approximately 2 years). During clinic visits, bloods and other samples, lung function, questionnaires, and other investigations will be used to measure how the drug is working.

A study to learn more about a combination of CFTR modulator medicines in people with Cystic Fibrosis (VX 18 445 104)

This study is being done to learn more about a new medication called ‘VX-445’ when taken in combination with tezacaftor and ivacaftor. All three of these medications are known as CFTR modulators. CFTR modulators work to treat the underlying genetic cause of cystic fibrosis. This combination of medication is being assessed in people with Cystic Fibrosis who are 12 years and older and who are heterozygous for the F508del mutation and a gating or residual function mutation. The study will investigate how well these medications work and to check that they are safe to use. It is the hope that this combination of medications will have positive effects in some people with Cystic Fibrosis Participants will be asked to come into the clinic at the recruitment centre 9 times over a 5 month period where a study assessment will take place (blood tests, questionnaires, lung function etc.).

A study of the combined effects of three CFTR modulator medicines in people with cystic fibrosis aged 12 and older (VX18-445-113)

In order to take part in this study you will already be taking part in the VX17-659-105 study, taking VX-659 with tezacaftor and ivacaftor. For this new study, you will switch to taking VX-445 instead of VX-659. Both combinations work in the same way. 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 long-term safety of VX-445 in combination with tezacaftor and ivacaftor. All three are CFTR modulators which means that they help the faulty CFTR protein 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. Everyone in the study will get the study medications. If you meet the criteria for being in the study, you will take VX-445, tezacaftor and ivacaftor in the morning (in 2 tablets), and ivacaftor in the evening (1 tablet). You will be asked to come into clinic around 12 times over the 22-month (nearly 2 years) participation period. During visits, the effect of the treatment will be measured with sample collection, lung function, ECG and other medical checks. You will also receive 14 telephone calls during the participation period, each lasting around half an hour. You do not have to come to clinics for these calls. This study is expected to recruit 400 participants worldwide.

A study to learn more about a combination of CFTR modulator medicines in people with Cystic Fibrosis

This study is being done to learn more about a new medication called ‘VX-445’ when taken in combination with tezacaftor and ivacaftor. All three of these medications are known as CFTR modulators. CFTR modulators work to treat the underlying genetic cause of cystic fibrosis. This combination of medication is being assessed in people with Cystic Fibrosis who are 12 years and older and who are homozygous for the F508del mutation. The study will investigate how well these medications work and to check that they are safe to use. It is the hope that this combination of medications will have positive effects in some people with Cystic Fibrosis. Participants will be asked to come into the clinic at the recruitment centre 7-8 times over a 5 month period where study assessments will take place (blood tests, questionnaires, lung function etc.).