• Genetic TherapyEnrolling

  Study of ARCT-032 in adults with cystic fibrosis who are not on CFTR modulator therapy. , protocol number Arcturus ARCT-032-02

  This study will test the safety, tolerability and effectiveness of ARCT-032, a nebulized therapy intended to deliver a new, correct copy of CFTR messenger RNA (mRNA) to cells.

  • Age:

    18 Years and Older

  • Mutation(s):

    No Mutation Requirement

  • FEV1% Predicted:

    40 to 100%

  • Number of Visits:

    8

  • Length of Participation:

    20 weeks

• Genetic TherapyEnrolling

  Study to evaluate KB407 in adults with cystic fibrosis , protocol number Krystal Bio KB407-02

  This study will evaluate safety and tolerability of ascending doses of nebulized KB407, a gene therapy intended to deliver a new, correct copy of the CFTR gene (DNA) to cells in adults with cystic fibrosis.

  • Age:

    18 Years and Older

  • Mutation(s):

    No Mutation Requirement

  • FEV1% Predicted:

    50 to 100%

  • Number of Visits:

    10

  • Length of Participation:

    3 months

• Genetic TherapyEnrolling

  Study to evaluate RCT2100 in adults with cystic fibrosis who are not eligible for/or not taking CFTR modulator therapy (Part 2) , protocol number ReCode RCT2100-101 Part 2 MAD

  This study will evaluate the safety and tolerability of RCT2100, an investigational inhaled messenger RNA (mRNA), in adults with CF who are not eligible for CFTR modulator therapy or who are not able to take CFTR modulatory therapy.

  • Age:

    18 Years and Older

  • Mutation(s):

    No Mutation Requirement

  • FEV1% Predicted:

    40 to 100%

  • Number of Visits:

    41

  • Length of Participation:

    10 months

• Genetic TherapyEnrolling

  Study to evaluate SP-101 in adults with cystic fibrosis who are ineligible for or cannot tolerate CFTR modulator therapy. , protocol number Spirovant CFAAV-001 US

  This study will evaluate the safety and tolerability of SP-101, an inhaled gene therapy, in adults with cystic fibrosis ages 18 to 65 who are ineligible for or cannot tolerate CFTR modulator therapy.

  • Age:

    18 Years to 65 Years

  • Mutation(s):

    No Mutation Requirement

  • FEV1% Predicted:

    50 to 100%

  • Number of Visits:

    11

  • Length of Participation:

    1 years

• Genetic TherapyEnrolling

  Study to evaluate SPL84 in adults with cystic fibrosis carrying the 3849 + 10 Kb C->T mutation , protocol number SpliSense SPL84-002

  This study will evaluate the safety, tolerability, effectiveness, and effect on the body of SPL84, an inhaled ASO, in adults with CF carrying the 3849 + 10 Kb C->T mutation.

  • Age:

    18 Years and Older

  • Mutation(s):

    One Copy F508del or No Copies F508del

  • FEV1% Predicted:

    40 to 90%

  • Number of Visits:

    13

  • Length of Participation:

    115 days

• Genetic TherapyEnrolling

  Study to evaluate VX-522 in adults 18 years and older with cystic fibrosis. , protocol number Vertex VX-522-001 MAD (VX21-522-001)

  This study will evaluate the safety and tolerability of VX-522, an investigational inhaled messenger RNA (mRNA) therapy, in adults with CF whose mutations are not responsive to CFTR modulator therapy.

  • Age:

    18 Years to 65 Years

  • Mutation(s):

    No Copies F508del

  • FEV1% Predicted:

    40 to 90%

  • Number of Visits:

    27

  • Length of Participation:

    8 months

• Genetic TherapyEnrolling

  Study of 4D-710 in Adults with Cystic Fibrosis , protocol number 4DMT 4D-710-C001 (Part 1 and 2)

  This study will test the safety and tolerability of 4D-710, an investigational gene therapy, in adults with CF who are not eligible for or are unable to tolerate CFTR modulator therapy.

  • Age:

    18 Years and Older

  • Mutation(s):

    No Mutation Requirement

  • FEV1% Predicted:

    50 to 90%

  • Number of Visits:

    11

  • Length of Participation:

    1 years

• Restore CFTR ProteinEnrolling

  Study of VX-828 in combination with Tezacaftor and Deutivacaftor in healthy participants and in adults with cystic fibrosis. , protocol number VX23-828-001 Cohort E

  Part E of this study will look at the safety and tolerability of VX-828 in combination with Tezacaftor and Deutivacaftor in adults with CF. The drug combination is intended to help CFTR function closer to normal. 

  • Age:

    18 Years and Older

  • Mutation(s):

    One Copy F508del

  • FEV1% Predicted:

    40% or greater

  • Number of Visits:

    12

  • Length of Participation:

    3 months

• Restore CFTR ProteinEnrolling

  Phase 3 study of VX-121/tezacaftor/deutivacaftor in children ages 2 to 5 with cystic fibrosis , protocol number Vertex VX21-121-105 Cohort B2

  This study is taking place at multiple care centers across the U.S. It will look at the safety and tolerability and how the body processes the drug VX-121/tezacaftor/deutivacaftor in participants with at least one triple-combination responsive CF mutation. 

  • Age:

    2 Years to 5 Years

  • Mutation(s):

    Mutation Requirement

  • FEV1% Predicted:

    No FEV1 Limit

  • Number of Visits:

    8

  • Length of Participation:

    32 weeks

• Anti-InflammatoryEnrolling

  Study to evaluate BI 1291583 in adults with bronchiectasis , protocol number BI 1397-0014 (Airtivity)

  This study will test the effectiveness, safety, and tolerability of BI 1291583, a drug intended to reduce inflammation in the lungs.

  • Age:

    18 Years and Older

  • Mutation(s):

    No Mutation Requirement

  • FEV1% Predicted:

    No FEV1 Limit

  • Number of Visits:

    9

  • Length of Participation:

    76 weeks

• Anti-InfectiveEnrolling

  Study to evaluate two antibiotic treatment approaches for the management of outpatient pulmonary exacerbations in children 3 to 18 years old. , protocol number STOP PEDS 2.0 RCT Main Cohort

  The STOP Peds study will evaluate the safety and effectiveness of two antibiotic treatment approaches for pulmonary exacerbations in children with CF.

  • Age:

    3 Years to 18 Years

  • Mutation(s):

    No Mutation Requirement

  • FEV1% Predicted:

    50% or greater

  • Number of Visits:

    6

  • Length of Participation:

    0 days

• Anti-InfectiveEnrolling

  Study of CMTX-101 in adults with cystic fibrosis chronically infected with Pseudomonas aeruginosa , protocol number Clarametyx CMTX101-P1-CT002 Part 2

  This study will test the safety and tolerability of a single dose of CMTX-101 delivered through an IV infusion. CMTX-101 is a drug intended to help treat infections in the lung.

  • Age:

    18 Years and Older

  • Mutation(s):

    No Mutation Requirement

  • FEV1% Predicted:

    35% or greater

  • Number of Visits:

    8

  • 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.