• Genetic TherapyEnrolling

  Study to evaluate SPL84 in adults with cystic fibrosis carrying the 3849 + 10 Kb C->T mutation , protocol number SpliSense SPL84-002 Phase 2a Cohort 4 (HEMT)

  Cohort 4 of 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 and who are stable on either Trikafta or Alyftrek.

  • Age:

    18 Years and Older

  • Mutation(s):

    One Copy F508del or No Copies F508del

  • FEV1% Predicted:

    40 to 80%

  • Number of Visits:

    18

  • Length of Participation:

    6 months

• Genetic TherapyClosed to Enrollment

  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

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.