Cystic Fibrosis (CF) MedDRA version: 20.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
Corbus Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Documentation of a CF diagnosis as evidenced by 1 or more clinical features consistent with the CF phenotype and 1 or more of the following criteria: a. Sweat chloride = 60 mEq/L by quantitative pilocarpine iontophoresis test b. Two known disease-causing mutations in the CFTR gene. 2. Twelve years of age or older at the time Informed Consent/Assent is signed. 3. Weight = 40 kg. 4. FEV1 = 40% predicted and =65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: 1. Severe or unstable CF at screening or Visit 1, such as: a. Change in dose, or initiation of any new chronic therapy for CF lung disease within 28 days before Visit 1 b. Treatment with any systemic corticosteroids > 10 mg per day prednisone or equivalent within 14 days before Visit 1 c. Actively listed on an organ transplant list or have had an organ transplant other than corneal transplant. 2. Significant diseases or conditions other than CF that may influence response to the study drug or safety, such as: a. Active hepatitis B or C infection b. Human immunodeficiency virus infection c. A history of cancer except basal cell carcinoma or in situ carcinoma of the cervix treated with apparent success with curative therapy = one year before Visit 1. 3. Subjects with a history of any seizure within the last 2 years. 4. Pregnant, trying to become pregnant or lactating female. 5. Current evidence of alcohol abuse (defined as 4 or more drinks per day on at least 4 days of the week) or history of abuse of illegal and/or legally prescribed drugs such as barbiturates, benzodiazepines, amphetamines, cocaine, or opioids during the 1 year before screening. 6. Any investigational agent within 30 days or five therapeutic half-lives of that agent whichever is longer, before Visit 1 7. Any of the following values for laboratory tests at screening: a. A positive pregnancy test b. Hemoglobin 2.5 x upper normal limit 8. Any other condition or concurrent medical therapy at screening or Visit 1 that causes the investigator to determine it is not safe for the subject to participate or that may influence response to study drug or interfere with study assessments.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy of lenabasum 20 mg twice per day (BID) compared to placebo in the treatment of cystic fibrosis (CF) by assessing the rate of pulmonary exacerbations (PEx) using primary definition of PEx;Secondary Objective: Efficacy: 1. To evaluate the efficacy of lenabasum 20 mg BID compared to placebo in the treatment of CF by assessing other efficacy endpoints 2. To evaluate the efficacy of lenabasum 5 mg BID compared to placebo in the treatment of CF Pharmacokinetic: 1. To evaluate steady state plasma concentrations of lenabasum 20 mg and 5 mg BID at the estimated time of trough concentration after a dose of lenabasum 2. To evaluate plasma concentrations of lenabasum 20 mg and 5 mg at the estimated time of peak concentration after the first dose 3. To evaluate metabolites of lenabasum 4. To develop population pharmacokinetic models of lenabasum exposure in CF subjects Safety: 1. To evaluate safety of lenabasum 20 mg BID and lenabasum 5 mg BID treatment and placebo treatment 2. To evaluate tolerability of lenabasum 20 mg BID and lenabasum 5 mg BID treatment;Primary end point(s): Rate of PEx using primary definition of PEx with lenabasum 20 mg BID, compared to placebo, during the treatment period;Timepoint(s) of evaluation of this end point: Visit 1 through study completion, up to 6 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Efficacy (lenabasum 20 mg BID): a. Event rate of PEx using secondary definition of PEx with lenabasum 20 mg BID compared to placebo b. Time to first new PEx using primary definition of PEx with lenabasum 20 mg BID compared to placebo c. Time to first PEx using secondary definition of PEx with lenabasum 20 mg BID compared to placebo d. Change from baseline in CFQ-R respiratory symptom domain with lenabasum 20 mg BID compared to placebo e. Change from baseline in FEV1 % predicted with lenabasum 20 mg BID compared to placebo Efficacy (lenabasum 5 mg BID): a. Rate of pulmonary exacerbations (PEx) using primary definition of PEx with lenabasum 5 mg BID compared to placebo, during the treatment period b. Event rate of PEx using secondary definition of PEx with lenabasum 5 mg BID compared to placebo c. Time to first new PEx using primary definition of PEx with lenabasum 5 mg BID compared to placebo d. Time to first PEx using secondary definition of PEx with lenabasum 5 mg BID compared to placebo e. Change from baseline in CFQ-R respiratory symptom domain with lenabasum 5 mg BID compared to placebo f. Change from baseline in FEV1 % predicted with lenabasum 5 mg BID compared to placebo Pharmacokinetics: 1. Estimated trough plasma concentrations of lenabasum 2. Estimated maximum plasma concentration (Cmax) of lenabasum 3. Metabolites of lenabasum Safety: 1. Treatment emergent adverse events (TEAEs) 2. Changes in vital signs, physical examination, blood and urine laboratory safety tests and electrocardiograms 3. Treatment discontinuations with lenabasum treatments compared to placebo;Timepoint(s) of evaluation of this end point: Efficacy and Safety endpoints: Visit 1 through study completions, up to 6 months; PK endpoints: Visit 1 through Visit 8. | — |
Countries
Austria, Belgium, Bulgaria, Canada, Czech Republic, France, Germany, Greece, Hungary, Italy, Netherlands, Poland, Portugal, Réunion, Romania, Russian Federation, Serbia, Slovakia, Spain, Sweden, United Kingdom, United States
Contacts
Public ContactRegulatory Services
TMC Pharma Services Ltd
Outcome results
None listed