• Restore CFTR ProteinCompleted with Results

  Phase 3 study of lumacaftor/ivacaftor (Orkambi®) in babies with two copies of the F508del CFTR mutation , protocol number Vertex VX-16-809-122

  This study evaluated the safety of lumacaftor/ivacaftor (Orkambi®) and its effect on the body in babies ages 1 to less than 2 years old who have cystic fibrosis and two copies of the F508del CFTR mutation.

  • Age:

    1 Years to 2 Years

  • Mutation(s):

    Two Copies F508del

  • FEV1% Predicted:

    No FEV1 Limit

  • Number of Visits:

    11

  • Length of Participation:

    38 weeks

• Restore CFTR ProteinCompleted with Results

  VX 809 and ivacaftor in people with CF who are aged 12 years and older and have two copies of the F508del-CFTR mutation (105) , protocol number Vertex VX-809-105

  This was an open-label follow-on study that evaluated the safety and efficacy of lumacaftor in combination with ivacaftor (Orkambi®) in people with CF, 12 years and older and who have two copies of the F508del genetic mutation.

  • Age:

    12 Years and Older

  • Mutation(s):

    Two Copies F508del

  • FEV1% Predicted:

    40 to 90%

  • Number of Visits:

    7

  • Length of Participation:

    96 weeks

• Restore CFTR ProteinCompleted with Results

  Vertex Lumacaftor (VX-809) and Ivacaftor in Children with CF aged 6 to 11 years and have two copies of the delF508 CFTR mutation , protocol number Vertex VX13-809-011b

  This was an open-label study designed to look at the safety of lumacaftor in combination with ivacaftor (Orkambi®). This study was for younger children with CF who have two copies of the F508del CFTR mutation.

  • Age:

    6 Years to 11 Years

  • Mutation(s):

    Two Copies F508del

  • FEV1% Predicted:

    No FEV1 Limit

  • Number of Visits:

    11

  • Length of Participation:

    27 weeks

• Restore CFTR ProteinCompleted with Results

  Phase 3 study of lumacaftor/ivacaftor in children with CF , protocol number Vertex VX15-809-115

  This study evaluated the safety and effectiveness of lumacaftor in combination with ivacaftor (Orkambi®) in people who have two copies of the F508del mutation.

  • Age:

    2 Years to 5 Years

  • Mutation(s):

    Two Copies F508del

  • FEV1% Predicted:

    40% or greater

  • Number of Visits:

    11

  • Length of Participation:

    32 weeks

• Restore CFTR ProteinCompleted with Results

  Phase 3 study of lumacaftor and ivacaftor in children with cystic fibrosis , protocol number Vertex VX-809-109

  This study evaluated the safety and effectiveness of lumacaftor and ivacaftor (Orkambi®) combination therapy in children with CF.

  • Age:

    6 Years to 11 Years

  • Mutation(s):

    Two Copies F508del

  • FEV1% Predicted:

    70 to 105%

  • Number of Visits:

    5

  • Length of Participation:

    24 weeks

• Restore CFTR ProteinCompleted with Results

  Phase 3 study of lumacaftor and ivacaftor in with cystic fibrosis with advanced lung disease , protocol number Vertex VX-809-106

  This study evaluated the safety of the drugs lumacaftor and ivacaftor (Orkambi®). This study was for people with CF who have two copies of the F508del CFTR mutation and have advanced lung disease.

  • Age:

    12 Years and Older

  • Mutation(s):

    Two Copies F508del

  • FEV1% Predicted:

    Less than 40%

  • Number of Visits:

    14

  • Length of Participation:

    52 weeks

• Restore CFTR ProteinCompleted with Results

  Vertex 809/770 in People with Cystic Fibrosis and F508del-CFTR mutation , protocol number Vertex VX-809-102

  This study looked at the safety and effectiveness of VX 809 (lumacaftor) when taken alone and when in combination with VX770 (ivacaftor) when given to people with CF who have either one or two copies of the F508del-CFTR mutation.

  • Age:

    18 Years and Older

  • Mutation(s):

    Two Copies F508del or One Copy F508del

  • FEV1% Predicted:

    40% or greater

  • Number of Visits:

    6

  • Length of Participation:

    2 months

• Restore CFTR ProteinCompleted with Results

  Vertex Lumacaftor (VX-809) and Ivacaftor in People with CF aged 12 years and older and have two copies of the delF508 CFTR mutation (TRANSPORT) , protocol number Vertex VX-809-104

  This was the second of two nearly identical Phase 3 trials looking at the safety and effectiveness of the study drug VX 809 (lumacaftor) in combination with ivacaftor (Kalydeco) in people with CF who are aged 12 years and older and have two copies of the F508del-CFTR mutation.

  • Age:

    12 Years and Older

  • Mutation(s):

    Two Copies F508del

  • FEV1% Predicted:

    40% or greater

  • Number of Visits:

    11

  • Length of Participation:

    28 weeks

• Restore CFTR ProteinCompleted with Results

  Vertex Lumacaftor (VX-809) and ivacaftor in people with CF who are aged 12 years and have two copies of the delF508 CFTR mutation (TRAFFIC) , protocol number Vertex VX-809-103

  This was the first of two nearly identical Phase 3 trials looking at the safety and effectiveness of the study drug VX 809 (lumacaftor) in combination with ivacaftor in people with CF who are aged 12 years and older and have two copies of the F508del-CFTR mutation.

  • Age:

    12 Years and Older

  • Mutation(s):

    Two Copies F508del

  • FEV1% Predicted:

    40% or greater

  • Number of Visits:

    10

  • Length of Participation:

    24 weeks

Studies in this tool are multi-center studies facilitated by the Cystic Fibrosis Therapeutics Development Network. For a complete list of cystic fibrosis related studies, visit www.clinicaltrials.gov.