• Genetic TherapyEnrolling

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

  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% or greater

  • Number of Visits:

    8

  • Length of Participation:

    6 months

• Restore CFTR ProteinEnrolling

  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

• Anti-InfectiveEnrolling

  Study to evaluate the standardizing of treatment for pulmonary exacerbations in children and adults with CF ages 6 and older. , protocol number STOP360-IP-22

  This study will look at pulmonary exacerbations, which are a worsening of respiratory symptoms, in people with CF who need to be treated with intravenous (IV) antibiotics. It will compare treatment with one antibiotic (a beta-lactam) to treatment with two antibiotics (tobramycin and a beta-lactam) to learn whether there is a difference in lung function and symptom improvement between the two groups. This study is for people ages 6 and older.

  • Age:

    6 Years and Older

  • Mutation(s):

    No Mutation Requirement

  • FEV1% Predicted:

    No FEV1 Limit

  • Number of Visits:

    3

  • Length of Participation:

    48 days

• Nutritional-GIEnrolling

  Study to evaluate ANG003 enzyme therapy in adults with cystic fibrosis who have pancreatic insufficiency. , protocol number Anagram ANG003-22-101

  This phase 1 study will evaluate the safety, tolerability, and effectiveness of a single dose of ANG003. One dose of the study drug will be given in place of a participant's usual pancreatic enzymes.

  • Age:

    18 Years and Older

  • Mutation(s):

    No Mutation Requirement

  • FEV1% Predicted:

    No FEV1 Limit

  • Number of Visits:

    4

  • Length of Participation:

    23 days

• ObservationalEnrolling

  Rare mutation cell collection (RARE) , protocol number RARE-OB-16

  This study is taking place at six regional care centers across the U.S. Researchers will collect and make available for study cells from people with rare CFTR mutations.

  • Age:

    12 Years and Older

  • Mutation(s):

    One Copy F508del or No Copies F508del

  • FEV1% Predicted:

    No FEV1 Limit

  • Number of Visits:

    1

  • Length of Participation:

    2 days

• OtherEnrolling

  Study of SPI-1005 in people with CF ages 18 and older , protocol number Sound Pharma SPI-3005-501.2

  This study will test the safety and tolerability of SPI-1005, a drug intended to prevent and treat hearing loss caused by aminoglycosides.

  • Age:

    18 Years and Older

  • Mutation(s):

    No Mutation Requirement

  • FEV1% Predicted:

    40% or greater

  • Number of Visits:

    6

  • Length of Participation:

    49 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.