• Restore CFTR ProteinClosed to Enrollment

  Phase 3 study of long-term ivacaftor in babies who have a CFTR gating mutation , protocol number Vertex VX-770-126

  This open-label study is taking place at multiple care centers across the U.S. It will look at the safety and effectiveness of long-term ivacaftor in babies who have a CFTR gating mutation.

  • Age:

    Less than 25 Months

  • Mutation(s):

    One Copy F508del or No Copies F508del

  • FEV1% Predicted:

    No FEV1 Limit

  • Number of Visits:

    18

  • Length of Participation:

    128 weeks

• Restore CFTR ProteinClosed to Enrollment

  Phase 3 study of ivacaftor in babies who have a CFTR gating mutation , protocol number Vertex VX15-770-124

  This two-part, open-label study is taking place at multiple care centers across the U.S. It will look at the safety and effectiveness of ivacaftor, as well as how the body processes the drug, in babies who have a CFTR gating mutation.

  • Age:

    Less than 13 Months

  • Mutation(s):

    One Copy F508del or No Copies F508del

  • FEV1% Predicted:

    No FEV1 Limit

  • Number of Visits:

    8

  • Length of Participation:

    24 weeks

• Restore CFTR ProteinCompleted with Results

  Roll-over study of ivacaftor in children with CF who have a CFTR gating mutation , protocol number Vertex VX-770-109

  This study evaluated the long-term safety of ivacaftor (Kalydeco®) in children with CF. This study was for children who have a CFTR gating mutation and received at least one dose of ivacaftor in the VX11-770-108 study.

  • Age:

    2 Years to 5 Years

  • Mutation(s):

    One Copy F508del or No Copies F508del

  • FEV1% Predicted:

    No FEV1 Limit

  • Number of Visits:

    7

  • Length of Participation:

    88 weeks

• Restore CFTR ProteinCompleted with Results

  Open-label study of ivacaftor in people with CF who have a non-G551D CFTR gating mutation , protocol number Vertex VX-770-112

  This study evaluated the long-term safety of ivacaftor (Kalydeco®) in participants who have a non-G551D CFTR gating mutation. This study was for people who had participated in one of the VX11-770-110, VX12-770-111, or VX12-770-113 studies.

  • Age:

    6 Years and Older

  • Mutation(s):

    One Copy F508del or No Copies F508del

  • FEV1% Predicted:

    No FEV1 Limit

  • Number of Visits:

    7

  • Length of Participation:

    2 years

• Restore CFTR ProteinCompleted with Results

  Phase 3 study of ivacaftor in babies 12-24 month old who have a CFTR gating mutation , protocol number Vertex VX15-770-124

  This study evaluated the safety of ivacaftor (Kalydeco®) as well as how the body processes the drug in babies who have at least one copy of a CFTR gating mutation.

  • Age:

    12 Months to 24 Months

  • Mutation(s):

    One Copy F508del or No Copies F508del

  • FEV1% Predicted:

    No FEV1 Limit

  • Number of Visits:

    8

  • Length of Participation:

    24 weeks

• Restore CFTR ProteinCompleted with Results

  Ivacaftor in People with CF age 2 to 5 years with a CFTR Gating Mutation , protocol number Vertex VX-770-108

  This was an open-label study designed to look at the safety and effectiveness of ivacaftor (Kalydeco®) in children with CF who had at least one copy of a CFTR gating mutation.

  • Age:

    2 Years to 5 Years

  • Mutation(s):

    One Copy F508del or No Copies F508del

  • FEV1% Predicted:

    No FEV1 Limit

  • Number of Visits:

    11

  • Length of Participation:

    32 weeks

• Restore CFTR ProteinCompleted with Results

  Ivacaftor in People with CF who have a Non G551D Gating Mutation (KONNECTION) , protocol number Vertex VX-770-111

  The purpose of this trial was to look at the safety and effectiveness of ivacaftor (Kalydeco) in people with cystic fibrosis (CF) who have a non-G551D cystic fibrosis transmembrane regulator (CFTR) gating mutation (any one of the following CFTR mutations: G178R, G551S, S549N, S549R, G970R, G1244E, S1251N, S1255P, or G1349D).

  • Age:

    6 Years and Older

  • Mutation(s):

    One Copy F508del or No Copies F508del

  • FEV1% Predicted:

    40 to 105%

  • Number of Visits:

    11

  • Length of Participation:

    40 weeks

• Restore CFTR ProteinCompleted with Results

  Ivacaftor in People with CF who have the R117H-CFTR mutation (KONDUCT) , protocol number Vertex VX-770-110

  This study looked at the safety and effectiveness of ivacaftor (Kalydeco) in people with CF who have the R117H-CFTR mutation.

  • Age:

    6 Years and Older

  • Mutation(s):

    One Copy F508del or No Copies F508del

  • FEV1% Predicted:

    40 to 105%

  • Number of Visits:

    8

  • Length of Participation:

    28 weeks

• Restore CFTR ProteinCompleted with Results

  VX-770 Expanded Access Program , protocol number Vertex VX11-770-901

  This open-label expanded access program (EAP) study was designed to provide ivacaftor to patients in critical medical need prior to commercial product availability.

  • Age:

    6 Years and Older

  • Mutation(s):

    One Copy F508del or No Copies F508del

  • FEV1% Predicted:

    Less than 40%

  • Number of Visits:

    1

  • Length of Participation:

    9 months

• Restore CFTR ProteinCompleted with Results

  Vertex 770 - a phase 2 trial evaluating the effect of VX- 770 on Lung Clearance Index , protocol number Vertex VX10-770-106

  This trial studied the effect of VX-770 (ivacaftor) on lung clearance index in people with CF ages 6 years and older with the G551D mutation and an FEV1 >90%.

  • Age:

    6 Years and Older

  • Mutation(s):

    One Copy F508del or No Copies F508del

  • FEV1% Predicted:

    90% or greater

  • Number of Visits:

    8

  • Length of Participation:

    19 weeks

• Restore CFTR ProteinCompleted with Results

  VX-770 in People with CF and G551D Mutation - Open Label Safety Study (PERSIST) , protocol number Vertex VX-770-105

  This study looked at the long-term safety of ivacaftor in people with CF and the G551D mutation. Subjects received ivacaftor for 96 weeks during this study.

  • Age:

    6 Years and Older

  • Mutation(s):

    No Mutation Requirement

  • FEV1% Predicted:

    No FEV1 Limit

  • Number of Visits:

    10

  • Length of Participation:

    2 years

• Restore CFTR ProteinCompleted with Results

  VX-770 Phase 2 study in People with CF and Genotype G551D , protocol number Vertex VX06-770-101

  This Phase 2 clinical study looked at the safety and tolerability of ivacaftor (VX-770) in people with CF and at least one allele with the G551D CFTR mutation.

  • Age:

    18 Years and Older

  • Mutation(s):

    One Copy F508del or No Copies F508del

  • FEV1% Predicted:

    40% or greater

  • Number of Visits:

    7

  • Length of Participation:

    56 days

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.