This study is a Phase 2, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of CTX-4430 administered once-daily for 48 weeks for treatment of CF.
This study is a Phase 2, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of CTX-4430 administered once-daily for 48 weeks for treatment of CF. A total of 195 pulmonary CF patients that meet all the inclusion and no exclusion criteria and provide written informed consent will be randomized to receive 50 mg CTX-4430, 100 mg CTX-4430, or placebo in a 1:1:1 ratio. Follow-up visits will be conducted approximately every 4 weeks from Week 4 to Week 52 (4 weeks after completion of treatment).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
200
Difference From Placebo in Absolute Change From Baseline in Forced Expiratory Volume in 1 Second Percent Predicted (ppFEV1)
Difference from Placebo in absolute change from Baseline at Week 48 was assessed for FEV1 percent predicted.
Time frame: Baseline, Week 48
Number of Pulmonary Exacerbations Through 48 Weeks
Rate of protocol-defined pulmonary exacerbations reported through the Week 48/Early Termination visit will be annualized where a year is defined by 52 weeks and will be analyzed using a negative binomial regression.
Time frame: Week 48
Hazard Ratio Pulmonary Exacerbation While in the Study
Hazard Ratio of pulmonary exacerbation versus placebo for all subjects. Pulmonary exacerbations are defined as treatment with oral, inhaled, or intravenous antibiotic(s) for ≥4 of symptoms/signs per the modified Fuchs criteria.
Time frame: Week 48
Subjects Without a Pulmonary Exacerbation While in the Study
Subjects who did not experience a protocol-defined pulmonary exacerbation during the study
Time frame: Week 48
Relative Change (Percent Change) From Baseline in ppFEV1
Percent change from Baseline for ppFEV1 at 48 weeks was assessed.
Time frame: Baseline, Week 48
Change From Baseline at 48 Weeks for Forced Vital Capacity Percent Predicted (FVC) and FEF25-75% (Forced Expiratory Flow During the Middle Half of the Forced Vital Capacity) Percent Predicted
Time frame: Baseline, Week 48
Change From Baseline for Specified Biomarkers
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University of Alabama
Birmingham, Alabama, United States
Providence Health and Services
Anchorage, Alaska, United States
Banner University of Arizona Medical Center
Tucson, Arizona, United States
Childrens Hospital Los Angeles
Los Angeles, California, United States
Pediatric Pulmonary and Cystic Fibrosis Clinic, Stanford University
Palo Alto, California, United States
University of California Davis Medical Center
Sacramento, California, United States
National Jewish Health
Denver, Colorado, United States
Yale University
New Haven, Connecticut, United States
University of Florida
Gainesville, Florida, United States
Central Florida Pulmonary Group
Orlando, Florida, United States
...and 65 more locations
Results were only calculated in subjects who had a verifiable result at the Baseline and Week 48 visits.
Time frame: Baseline, Week 48
Change From Baseline for C-reactive Protein (Hs-CRP)
Results were only calculated in subjects who had a verifiable result at the Baseline and Week 48 visits.
Time frame: Baseline, Week 48