• Genetic TherapyEnrolling

  Study of 4D-710 in Adults with Cystic Fibrosis , protocol number 4DMT 4D-710-C001

  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 100%

  • Number of Visits:

    11

  • Length of Participation:

    2 years

• 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 inhaled AR-501 in healthy adults and adults with cystic fibrosis and Pseudomonas aeruginosa , protocol number Aridis AR-501-001 MAD cohorts

  This study is testing inhaled AR-501, a drug intended to treat infections in the lung. The study will measure the drug’s safety, how well it works, and how the body processes it. Multiple doses of AR-501 will be tested in both healthy adults and adults with cystic fibrosis who are infected with Pseudomonas aeruginosa to find the best dose.

  • Age:

    18 Years and Older

  • Mutation(s):

    No Mutation Requirement

  • FEV1% Predicted:

    45% or greater

  • Number of Visits:

    11

  • Length of Participation:

    6 weeks

• Nutritional-GIEnrolling

  Study to evaluate Creon in adults with cystic fibrosis or chronic pancreatitis , protocol number AbbVie M21-432

  This study will look at the symptoms of exocrine pancreatic insufficiency (EPI) in adults with cystic fibrosis or chronic pancreatitis treated with Creon with an alternate source of active drug. Creon is a drug approved to treat EPI. 

  • Age:

    18 Years and Older

  • Mutation(s):

    No Mutation Requirement

  • FEV1% Predicted:

    No FEV1 Limit

  • Number of Visits:

    3

  • Length of Participation:

    142 days

• ObservationalEnrolling

  Study to evaluate the effects of CFTR modulators in infants and young children (BEGIN Part A) , protocol number BEGIN-OB-19 Part A

  This two-part observational study will look at the effects of CFTR modulators on growth in young children with CF. These drugs are intended to help CFTR protein function closer to normal.

  • Age:

    Less than 5 Years

  • Mutation(s):

    Mutation Requirement

  • FEV1% Predicted:

    62% or greater

  • Number of Visits:

    6

  • Length of Participation:

    3 years

• 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.