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Genetic Therapy Closed to Enrollment

Study to evaluate SPL84 in adults with cystic fibrosis carrying the 3849 + 10 Kb C->T mutation (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.

 

Study participants will receive either the study drug (SPL84), or a placebo by inhalation once a week for 9 weeks. SPL84 is an inhaled antisense oligonucleotide (ASO), a small molecule that helps fix problems with the CFTR RNA caused by splice mutations to allow cells to make functional CFTR protein.

 

This is a multiple ascending dose (MAD) study. Three doses are being evaluated. The study starts with the lowest dose cohort and once that is complete moves on to the next higher dose. In this study, researchers will assess for tolerability, safety and effect by monitoring a participant’s health. 

 

This study may require blood draws, lung function testing, electrocardiograms, and/or other measurements.

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    18 Years and Older

  • Mutation(s):

    One Copy F508del or No Copies F508del

  • FEV1% Predicted:

    40 to 90%

For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.

Other Primary Eligibility Criteria

Potential participants must have at least one copy of the 3849 + 10 Kb C ->T Mutation

Study Design

  • Study Type: ?more info

    Interventional

  • Randomized Study: ?more info

    Yes

  • Placebo Controlled: ?more info

    Yes

  • Length of Participation:

    115 days

  • Number of Study Visits:

    13

Additional Information

  • Phase: ?more info

    Phase Two

  • Study Sponsor: ?more info

    SpliSense

  • Study Drugs:

    SPL84

Study Sites

  • Closed to Enrollment

    California

    Center for Cystic Fibrosis at Keck Medical Center of USC, Los Angeles, CA 90033

  • Closed to Enrollment

    Massachusetts

    Boston Children's Hospital, Brigham & Women's Hospital, Boston, MA 02115

  • Closed to Enrollment

    Ohio

    Nationwide Children's Hospital, Columbus, OH 43205

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    18 Years and Older

  • Mutation(s):

    One Copy F508del or No Copies F508del

  • FEV1% Predicted:

    40 to 90%

For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.

Other Primary Eligibility Criteria

Potential participants must have at least one copy of the 3849 + 10 Kb C ->T Mutation

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