VERTEX 809 (LUMACAFTOR) STUDY (PHASE 3)

Study ID number: VX12-809-104 
Official Title: A Phase 3, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
Therapeutic Category: CFTR Modulator

VX-809 (Lumacaftor) and VX-770 (Ivacaftor) are two different but complementary drugs that may help correct some of the irregularities or deformities that the gene mutation causes. VX-809 and VX-770 were being studied together to see if they can help decrease some of the effects caused by CF. They are designed to help the mutated chloride channel work more effectively which could alleviate the cycle of mucus plugging, infection, and inflammation in the lungs caused by CF.

This was a Phase 3 study to learn about safety/tolerability and effects of VX-809 when given in combination with VX-770 to subjects with CF who are homozygous for the deltaF508 mutation (have 2 copies of the deltaF508 mutation) . VX-809 and VX-770 were investigated in how they may change your lung function (FEV1), weight, quality of life, and frequency of pulmonary exacerbations (lung flareups), etc over a 6 month treatment period.  

The study was completed and proceeded to an open label extension study (where all eligible participants to enroll were given drug)

Source: ClinicalTrials. gov:  https://clinicaltrials.gov/ct2/show/NCT01807949

Publications related to the study:

https://www.ncbi.nlm.nih.gov/pubmed/30587335?dopt=Abstract

https://www.ncbi.nlm.nih.gov/pubmed/27298017?dopt=Abstract

https://www.ncbi.nlm.nih.gov/pubmed/25981758?dopt=Abstract

OPEN LABEL EXTENSION STUDY:
Study ID number: VX12-809-105 
Official Title: A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation
Therapeutic Category: CFTR Modulator

This was an open label Phase 3 study to learn about the long term  safety/tolerability and effects of VX-809 when given in combination with VX-770 to subjects with CF who have previously enrolled and completed selected protocols. VX-809 and VX-770 were investigated in how they may change your lung function (FEV1), weight, quality of life, and frequency of pulmonary exacerbations (lung flareups), etc over a 96 week/~2 years treatment period. 

This drug was not effective for participants that were heterozygous for the delta F508 mutation. Therefore this was not approved for the heterozygous group. 

The study was completed and the drug was first approved for use on delta F508 homozygous CF patients on July 2015 in the United States. It was approved in Canada on January 2016. It is currently on the market as Orkambi.

Source: ClinicalTrials. gov:  https://clinicaltrials.gov/ct2/show/NCT01931839

Publications related to the study:

https://www.ncbi.nlm.nih.gov/pubmed/28011037?dopt=Abstract

 

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