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

Details

Therapeutic category
Restore CFTR Function
Trial status
Completed Participating Centres
Phase
Phase III

Full title

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 Gating or Residual Function Mutation (F/G and F/RF Genotypes)

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.).
Trial Reference Number
124417
Trial type
Medication
Intervention
CFTR Modulators
Recruitment target
250
Last edited date
23/09/2020
CF sponsor
Vertex Pharmaceuticals Incorporated
CF sponsor type
Commercial

Who can take part?

Top inclusion criteria
  • Heterozygous for F508del and either a gating or residual function mutation
  • 12 Years and older
  • FEV1 value ≥40% and ≤90%
Top exclusion criteria
  • Clinically significant cirrhosis with or without portal hypertension
  • Lung infection

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