This is a Phase 2 multicenter, double-blind, randomized, placebo-controlled study assessing the efficacy and safety of lenabasum for the treatment of cystic fibrosis in patients 12 years of age or older. Approximately 415 subjects will be enrolled in this study at about 100 sites in North America, and Europe. The planned duration of treatment with study drug is 28 weeks. Study drug will be lenabasum 20 mg BID, lenabasum 5 mg BID, and placebo in a 2:1:2 ratio.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
447
Subjects will receive lenabasum 20 mg twice daily.
Subjects will receive lenabasum 5 mg twice daily.
Subjects will receive placebo twice daily.
Pulmonary Exacerbation (PEx) Rate Over 28 Weeks
Rate of PEx using the primary PEx definition with lenabasum 20 mg BID compared to placebo, during the treatment period. An primary PEx is defined on the physician's decision to treat with oral, intraveneous or inhaled antibiotics in the presence 4/12 Fuch's criteria (Change in sputum, new/increased hemoptysis, increased cough, increased dyspnea, malaise, fatigue/lethargy, Temperature greater than 38C, weight loss, sinus pain, change in sinus discharge, change in exam of chest, decrease in FEV1 of more than 10%, radiographic change). This excludes prophylactic antibiotics. A new PEx is a one that occurs at 28 days after the previous PEx.
Time frame: 28 weeks (Baseline Day 0 to Week 28)
Pulmonary Exacerbation (PEx) Rate
Event rate of PEx using the secondary PEx definition with lenabasum 20 mg BID compared to placebo. The secondary definition of a PEx is based on the physician's diagnosis of pulmonary exacerbation and commencement of new oral, intravenous, or inhaled antibiotics. A new PEx is defined one that starts 28 or more days after the previous confirmed PEx. The PEx rate is calculated as the number of PEx/28 weeks
Time frame: 28 weeks (Baseline Day 0 to Week 28)
Time to First New Pulmonary Exacerbation (PEx)
Time to first new PEx using the primary PEx definition with lenabasum 20 mg BID compared to placebo. An primary PEx is defined on the physician's decision to treat with oral, intraveneous or inhaled antibiotics in the presence 4/12 Fuch's criteria (Change in sputum, new/increased hemoptysis, increased cough, increased dyspnea, malaise, fatigue/lethargy, Temperature greater than 38C, weight loss, sinus pain, change in sinus discharge, change in exam of chest, decrease in FEV1 of more than 10%, radiographic change). This excludes prophylactic antibiotics. A new PEx is a one that occurs at 28 days after the previous PEx. The rate is calculate over a 28 week period from visit 1 to week 28 visit
Time frame: 28 weeks (Baseline Day 0 to Week 28)
Pulmonary Exacerbation (PEx)
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Phoenix Children's Hospital
Phoenix, Arizona, United States
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States
Miller Children's Hospital
Long Beach, California, United States
National Jewish Health
Denver, Colorado, United States
University of Miami
Miami, Florida, United States
Central Florida Pulmonary Group, PA
Orlando, Florida, United States
USF Center for Advance Lung Disease
Tampa, Florida, United States
Emory Children's Center
Atlanta, Georgia, United States
Northwestern Memorial Hospital
Chicago, Illinois, United States
The Cystic Fibrosis Institute
Glenview, Illinois, United States
...and 95 more locations
Time to first PEx using the secondary PEx definition with lenabasum 20 mg BID compared to placebo. The secondary definition of a PEx is based on the physician's diagnosis of pulmonary exacerbation and commencement of new oral, intravenous, or inhaled antibiotics. A new PEx is defined one that starts 28 or more days after the previous confirmed PEx.
Time frame: 28 weeks (Baseline Day 0 to Week 28)
CFQ-R Respiratory Symptom Domain
Cystic Fibrosis Questionnaire - Revised measures change from baseline in CFQ-R respiratory symptom domain with lenabasum compared to placebo. Subjects \>/= 14 years of age. 5 distinct 4-point Likert scales (e.g., always/often/ sometime/never) Scores for each HRQoL domain; after recoding, each item is summed to generate a domain score and standardized. Scores range from 0 to 100, with higher scores indicating better health.
Time frame: 28 weeks (Change from Baseline Day 0 to Week 28)
FEV1 % Predicted
Change from baseline to week 28 in Forced Expiratory Volume in 1 second (FEV1) expressed as a percentage of a normal range. A lower percentage FEV1 is indicative of decrease in lung functionality. The changes observed from baseline to week 28 for lenabasum will be compared with those observed for placebo treated participants.
Time frame: 28 weeks (Change from Baseline Day 0 to Week 28)