• Restore CFTR FunctionEnrolling

  Study of Trikafta® in people with CF ages 12 years and older who have partial function CFTR mutations , protocol number Sorscher Partial Function Sub-Study

  This study will look at the effectiveness of Trikafta^®, a drug intended to help CFTR function closer to normal. This study is for people ages 12 and older who have two CFTR mutations not approved for Trikafta^® and evidence of partial CFTR function. Partial function is defined as a sweat chloride level < 80 mmol/L and/or pancreatic sufficiency (no pancreatic enzyme supplement therapy).

  • Age:

    12 Years and Older

  • Mutation(s):

    No Copies F508del

  • FEV1% Predicted:

    No FEV1 Limit

  • Number of Visits:

    5

  • Length of Participation:

    2 months

• Restore CFTR FunctionEnrolling

  Study of Trikafta® in people with CF ages 12 years and older who have an N1303K CFTR mutation , protocol number N1303K Sub-Study

  This study will look at the effectiveness of Trikafta, a drug intended to help CFTR function closer to normal. This study is for people ages 12 and older who have two CFTR mutations not approved for Trikafta^® and a least one of these mutations must be N1303K.

  • Age:

    12 Years and Older

  • Mutation(s):

    No Copies F508del

  • FEV1% Predicted:

    No FEV1 Limit

  • Number of Visits:

    5

  • Length of Participation:

    86 days

• Restore CFTR FunctionClosed to Enrollment

  Study of the triple-combination modulator, elexacaftor/tezacaftor/ivacaftor, in children 2-5 years old with cystic fibrosis (Part B) , protocol number VX20-445-111 PART B

  This study will look at the safety and effectiveness of the triple-combination therapy, elexacaftor/tezacaftor/ivacaftor (Trikafta®), in children ages 2-5 years old with CF. These drugs are intended to help CFTR protein function closer to normal.

  • Age:

    2 Years to 5 Years

  • Mutation(s):

    Two Copies F508del or One Copy F508del

  • FEV1% Predicted:

    No FEV1 Limit

  • Number of Visits:

    9

  • Length of Participation:

    32 weeks

• Restore CFTR FunctionClosed to Enrollment

  Study of the triple-combination modulator, elexacaftor/tezacaftor/ivacaftor, in children 2-5 years old with cystic fibrosis (Part A) , protocol number Vertex VX20-445-111 Part A

  This study will look at the safety and effectiveness of the triple-combination therapy, elexacaftor/tezacaftor/ivacaftor (Trikafta®), in children ages 2-5 years old with CF. These drugs are intended to help CFTR protein function closer to normal.

  • Age:

    2 Years to 5 Years

  • Mutation(s):

    Two Copies F508del or One Copy F508del

  • FEV1% Predicted:

    No FEV1 Limit

  • Number of Visits:

    5

  • Length of Participation:

    53 days

• Restore CFTR FunctionCompleted with Results

  Study of VX-445 Triple Combination in Teens and Adults With Cystic Fibrosis (CF) Who Have One Copy of F508del and One Copy of a Gating or Residual Function Mutation , protocol number Vertex VX18-445-104

  This study evaluated the safety and effectiveness of VX-445 (elexacaftor), tezacaftor, and ivacaftor in combination called TRIKAFTA®. This study was for people with CF 12 years and older with one copy of the F508del mutation and one copy of a gating or residual function mutation.

  • Age:

    12 Years and Older

  • Mutation(s):

    One Copy F508del

  • FEV1% Predicted:

    40 to 90%

  • Number of Visits:

    8

  • Length of Participation:

    20 weeks

• Restore CFTR FunctionCompleted with Results

  Study of VX-445 triple combination in children 6-11 years old with cystic fibrosis , protocol number Vertex VX18-445-106

  This study evaluated the safety and effectiveness of the triple combination modulator therapy, elexacaftor/tezacaftor/ivacaftor (Trikafta®) in children ages 6-11 years with CF, who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation.

  • Age:

    6 Years to 11 Years

  • Mutation(s):

    Two Copies F508del or One Copy F508del

  • FEV1% Predicted:

    40% or greater

  • Number of Visits:

    9

  • Length of Participation:

    32 weeks

• Restore CFTR FunctionCompleted with Results

  Study of VX-445 plus ivacaftor and tezacaftor in people with CF who have two copies of the F508del CFTR mutation , protocol number Vertex VX17-445-103

  This study evaluated the effectiveness and safety of the CFTR modulator drug VX-445 (elexacaftor) in combination with ivacaftor and tezacaftor. This triple combination is known as Trikafta™. This study was for people with CF ages 12 and older who have two copies of the F508del CFTR mutation and were already taking tezacaftor plus ivacaftor.

  • Age:

    12 Years and Older

  • Mutation(s):

    Two Copies F508del

  • FEV1% Predicted:

    40 to 90%

  • Number of Visits:

    7

  • Length of Participation:

    10 weeks

• Restore CFTR FunctionCompleted with Results

  Study of effectiveness and safety of elexacaftor/tezacaftor/ivacaftor in people with CF who have one copy of the F508del CFTR mutation , protocol number Vertex VX17-445-102

  This study evaluated the effectiveness and safety of the CFTR modulator drug VX-445 (elexacaftor) in combination with tezacaftor and ivacaftor (ELX/TEZ/IVA). This triple combination drug is known as Trikafta™. This study was for people with cystic fibrosis ages 12 and older with one copy of the F508del CFTR mutation and one copy of a minimal function mutation.

  • Age:

    12 Years and Older

  • Mutation(s):

    One Copy F508del

  • FEV1% Predicted:

    40 to 90%

  • Number of Visits:

    10

  • Length of Participation:

    32 weeks

• Restore CFTR FunctionCompleted with Results

  Phase 3 study of VX-659 triple combination drug in people with CF ages 12 years and older who have two copies of the F508del mutation , protocol number VX17-659-103

  This study evaluated the safety and effectiveness of the drug VX-659 in combination with tezacaftor and ivacaftor in people with cystic fibrosis ages 12 and older with two copies of the F508del CFTR mutation.

  • Age:

    12 Years and Older

  • Mutation(s):

    Two Copies F508del

  • FEV1% Predicted:

    40 to 90%

  • Number of Visits:

    7

  • Length of Participation:

    10 weeks

• Restore CFTR FunctionCompleted with Results

  Phase 3 study of VX-659 triple combination drug in people with CF 12 years and older who have one copy of the F508del mutation and one copy of a minimal function mutation , protocol number VX17-659-102

  This study evaluated the safety and effectiveness of the drug VX-659 in combination with tezacaftor and ivacaftor in people with cystic fibrosis ages 12 and older with one copy of the F508del CFTR mutation and one copy of a minimal function mutation.

  • Age:

    12 Years and Older

  • Mutation(s):

    One Copy F508del

  • FEV1% Predicted:

    40 to 90%

  • Number of Visits:

    10

  • Length of Participation:

    32 weeks

• Restore CFTR FunctionCompleted with Results

  A study to evaluate tezacaftor/ivacaftor in adults with cystic fibrosis and two copies of the F508del mutation , protocol number Vertex VX16-661-114

  This study evaluated the safety, effectiveness, and tolerability of the drug tezacaftor/ivacaftor (Symdeko®). This study was for people with CF ages 12 and older with two copies of the F508del CFTR mutation who had been taken off lumacaftor/ivacaftor (Orkambi®) due to respiratory side effects.

  • Age:

    12 Years and Older

  • Mutation(s):

    Two Copies F508del

  • FEV1% Predicted:

    25 to 90%

  • Number of Visits:

    5

  • Length of Participation:

    84 days

• Restore CFTR FunctionCompleted with Results

  Phase 2 study of VX-659 combination drug in adults with cystic fibrosis , protocol number Vertex VX16-659-101

  This study evaluated the safety, tolerability and effectiveness of the drug VX-659 in combination with tezacaftor/ivacaftor (Symdeko®) in people who either have two copies of the F508del CFTR mutation or have one copy of F508del and one copy of a minimal function 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:

    8 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.