• 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

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.