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Clinical Trial Finder
Genetic Therapy Enrolling
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.
Email a research coordinator to express interest in participating in this study.
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 a more comprehensive list of eligibility criteria and details on this study, 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:
Study Sites
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Enrolling Soon
Alabama
University of Alabama at Birmingham, Birmingham, AL 35294
Contact
Heather Hathorne
Phone: +1 (205) 638-9568
Email: hyhathorne@uabmc.edu
-
Enrolling Soon
California
University of California San Diego, La Jolla, CA 92093
Contact
Jenna Mielke
Phone: +1 (619) 471-0822
Email: jmielke@health.ucsd.edu
-
Enrolling Soon
California
Center for Cystic Fibrosis at Keck Medical Center of USC, Los Angeles, CA 90033
Contact
Lynn Fukushima
Phone: +1 (323) 409-5383
Email: lynn.fukushima@med.usc.edu
-
Enrolling Soon
Colorado
National Jewish Health, Denver, CO 80206
Contact
Alix Wilson
Phone: +1 (303) 270-2517
Email: wilsona@njhealth.org
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Enrolling Soon
Massachusetts
Boston Children's Hospital, Brigham & Women's Hospital, Boston, MA 02115
Contact
Robert Fowler
Phone: +1 (617) 355-1834
Email: Robert.fowler@childrens.harvard.edu
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Enrolling Soon
Ohio
Nationwide Children's Hospital, Columbus, OH 43205
Contact
Diana Gilmore
Phone: +1 (614) 722-4752
Email: diana.gilmore@nationwidechildrens.org
Email a research coordinator to express interest in participating in this study.
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 a more comprehensive list of eligibility criteria and details on this study, 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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