Safety, Tolerability, Pharmacokinetics, and Efficacy of JBT-101 (Lenabasum) in Cystic Fibrosis

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and efficacy of JBT-101 in adult subjects with cystic fibrosis (CF).

Detailed Description
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An interventional, double-blind, randomized, placebo-control design will be used to test safety, tolerability, pharmacokinetics, and efficacy of JBT-101 in 70 subjects ≥ 18 and < 65 years of age with documented cystic fibrosis. There will up a screening period of up to 28 days, 84 days treatment period, and 28 days follow-up.

Official Title
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A Phase 2, Double-blind, Randomized, Placebo-controlled Multicenter Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of JBT-101 in Cystic Fibrosis

Conditions
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Cystic Fibrosis

Intervention / Treatment
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* Drug: JBT-101 (lenabasum)
* Other: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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: Sweat chloride equal to or greater than 60 mEq/L by quantitative pilocarpine iontophoresis test; Two well-characterized mutations in the CFTR gene FEV1 ≥ 40% predicted corrected Stable treatment of CF for 14 days before Visit 1 Exclusion Criteria: Severe or unstable CF, such as: Intravenous antibiotic treatment within 14 days before Visit 1 Treatment with any corticosteroids > 10 mg per day or > 20 mg every other day oral prednisone or equivalent within 14 days before Visit 1 Any one of the following values for laboratory tests at Screening: A positive pregnancy test (or at Visit 1); Hemoglobin < 10 g/dL Neutrophils < 1.0 x 10 9/L Platelets < 75 x 10 9/L Creatinine clearance < 50 ml/min according to modified Cockcroft-Gault equation Serum transaminases > 2.5 x upper normal limit Total bilirubin ≥ 1.5 x upper limit of normal Any other condition that, in the opinion of the Principal Investigator, is clinically significant and may put the subject at greater safety risk, influence response to study product, or interfere with study assessments.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: JBT101 (lenabasum) 1 mg<br>JBT-101 1 mg once a day on Days 1-28 | Drug: JBT-101 (lenabasum)<br>* Subjects will receive JBT-101 1 mg qd or JBT-101 5 mg qd on Days 1-28. Subjects will receive either JBT-101 20 mg q am (with placebo q pm) or JBT-101 20 mg twice a day on Days 29-84.<br>|
| Experimental: JBT-101 (lenabasum) 5 mg<br>JBT-101 5 mg once a day on Days 1-28 | Drug: JBT-101 (lenabasum)<br>* Subjects will receive JBT-101 1 mg qd or JBT-101 5 mg qd on Days 1-28. Subjects will receive either JBT-101 20 mg q am (with placebo q pm) or JBT-101 20 mg twice a day on Days 29-84.<br>|
| Placebo Comparator: Placebo<br>Placebo once a day on Days 1-28. | Other: Placebo<br>* Subjects will receive placebo once a day on Days 1-28, placebo q pm on Days 29-84 (with JBT-101 20 mg q am), or placebo bid on Days 29-84.<br>|
| Experimental: JBT-101 (lenabasum) 20 mg QD<br>JBT-101 20 mg once a day on Days 29-84. | Drug: JBT-101 (lenabasum)<br>* Subjects will receive JBT-101 1 mg qd or JBT-101 5 mg qd on Days 1-28. Subjects will receive either JBT-101 20 mg q am (with placebo q pm) or JBT-101 20 mg twice a day on Days 29-84.<br>|
| Experimental: JBT-101 (lenabasum) 20 mg BID<br>JBT-101 20 mg twice daily on Days 29-84. | Drug: JBT-101 (lenabasum)<br>* Subjects will receive JBT-101 1 mg qd or JBT-101 5 mg qd on Days 1-28. Subjects will receive either JBT-101 20 mg q am (with placebo q pm) or JBT-101 20 mg twice a day on Days 29-84.<br>|
| Placebo Comparator: Placebo BID<br>Placebo twice daily on Days 29-84. | Other: Placebo<br>* Subjects will receive placebo once a day on Days 1-28, placebo q pm on Days 29-84 (with JBT-101 20 mg q am), or placebo bid on Days 29-84.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Treatment Emergent Adverse Events. |  | 84 days of treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| JBT-101 (Lenabasum) Plasma Concentrations on Day 84 | Plasma concentrations were reported for the lenabasum 20 mg QD, 20 mg BID, and placebo groups only, at Day 84. | Day 84 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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JBT-101, Lenabasum, Cystic Fibrosis