• 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 inhaled RSP-1502 in adults with cystic fibrosis and Pseudomonas aeruginosa , protocol number Respirion RESPIR-102

  This study will look at the safety of inhaled RSP-1502, a drug intended to treat infections in the lung. RSP-1502, which includes the antibiotic tobramycin, will be compared to tobramycin alone. The study will measure the drug's safety, how well it works, and how the body processes it. Four doses of RSP-1502 will be tested to find the best dose. 

  • Age:

    18 Years and Older

  • Mutation(s):

    No Mutation Requirement

  • FEV1% Predicted:

    40 to 90%

  • Number of Visits:

    5

  • Length of Participation:

    28 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

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

  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 6 Years

  • Mutation(s):

    Mutation Requirement

  • FEV1% Predicted:

    No FEV1 Limit

  • Number of Visits:

    6

  • Length of Participation:

    2 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.