Cystic Fibrosis
Conditions
Keywords
JBT-101, Lenabasum, Cystic Fibrosis
Brief summary
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
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.
Interventions
DRUGJBT-101 (lenabasum)
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.
OTHERPlacebo
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.
Sponsors
Corbus Pharmaceuticals Inc.
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
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: 1. Sweat chloride equal to or greater than 60 mEq/L by quantitative pilocarpine iontophoresis test; 2. 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: 1. Intravenous antibiotic treatment within 14 days before Visit 1 2. 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: 1. A positive pregnancy test (or at Visit 1); 2. Hemoglobin \< 10 g/dL 3. Neutrophils \< 1.0 x 10\ 9/L 4. Platelets \< 75 x 10\ 9/L 5. Creatinine clearance \< 50 ml/min according to modified Cockcroft-Gault equation 6. Serum transaminases \> 2.5 x upper normal limit 7. 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of Participants With Treatment Emergent Adverse Events. | 84 days of treatment |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| JBT-101 (Lenabasum) Plasma Concentrations on Day 84 | Day 84 | Plasma concentrations were reported for the lenabasum 20 mg QD, 20 mg BID, and placebo groups only, at Day 84. |
Countries
Belgium, France, Germany, Italy, Poland, United Kingdom, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| JBT101 1 mg QD, 20 mg QD, or 20 mg BID JBT-101: 1 mg once a day on Days 1-28 and either 20 mg QD or 20 mg BID on Days 29-84. | 26 |
| JBT-101 5 mg QD,20 mg QD, or 20 mg BID JBT-101: 5 mg once a day on Days 1-28 and either 20 mg QD or 20 mg BID on Days 29-84. | 24 |
| Placebo QD,20 mg QD, 20 mg BID, or Placebo BID Placebo: Subjects received placebo on Days 1 - 28 and either 20 mg QD, 20 mg BID, or Placebo on Days 29-84. | 35 |
| Total | 85 |
Baseline characteristics
| Characteristic | JBT101 1 mg QD, 20 mg QD, or 20 mg BID | JBT-101 5 mg QD,20 mg QD, or 20 mg BID | Placebo QD,20 mg QD, 20 mg BID, or Placebo BID | Total |
|---|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 1 Participants | 2 Participants | 3 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 26 Participants | 23 Participants | 33 Participants | 82 Participants |
| Age, Continuous | 26.9 years STANDARD_DEVIATION 7.66 | 28.8 years STANDARD_DEVIATION 10.4 | 29.2 years STANDARD_DEVIATION 8.55 | 28.4 years STANDARD_DEVIATION 8.81 |
| BMI | 22.735 kg/m^2 STANDARD_DEVIATION 3.8582 | 22.976 kg/m^2 STANDARD_DEVIATION 3.4182 | 22.608 kg/m^2 STANDARD_DEVIATION 3.4672 | 22.751 kg/m^2 STANDARD_DEVIATION 3.5381 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 2 Participants | 0 Participants | 2 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 26 Participants | 22 Participants | 35 Participants | 83 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Height | 171.30 cm STANDARD_DEVIATION 9.724 | 167.75 cm STANDARD_DEVIATION 9.192 | 167.82 cm STANDARD_DEVIATION 8.866 | 168.86 cm STANDARD_DEVIATION 9.259 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants | 0 Participants | 1 Participants | 2 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) White | 25 Participants | 24 Participants | 33 Participants | 82 Participants |
| Sex: Female, Male Female | 9 Participants | 12 Participants | 18 Participants | 39 Participants |
| Sex: Female, Male Male | 17 Participants | 12 Participants | 17 Participants | 46 Participants |
| Weight | 66.98 kg STANDARD_DEVIATION 14.648 | 64.71 kg STANDARD_DEVIATION 11.032 | 63.81 kg STANDARD_DEVIATION 11.806 | 65.03 kg STANDARD_DEVIATION 12.467 |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk | EG005 affected / at risk |
|---|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 26 | 0 / 24 | 0 / 35 | 0 / 30 | 0 / 28 | 0 / 23 |
| other Total, other adverse events | 13 / 26 | 13 / 24 | 15 / 35 | 21 / 30 | 19 / 28 | 14 / 23 |
| serious Total, serious adverse events | 1 / 26 | 0 / 26 | 2 / 37 | 3 / 30 | 2 / 28 | 1 / 23 |
Outcome results
Primary
Number of Participants With Treatment Emergent Adverse Events.
Time frame: 84 days of treatment
Population: These adverse events are based on subjects who entered Treatment Period 2 (n=81). The tabulation of adverse events includes all TEAEs.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| JBT101 1 mg QD | Number of Participants With Treatment Emergent Adverse Events. | 14 Participants |
| JBT-101 5 mg QD | Number of Participants With Treatment Emergent Adverse Events. | 13 Participants |
| Placebo QD | Number of Participants With Treatment Emergent Adverse Events. | 15 Participants |
| JBT-101 20 mg QD | Number of Participants With Treatment Emergent Adverse Events. | 21 Participants |
| JBT-101 20 mg BID | Number of Participants With Treatment Emergent Adverse Events. | 19 Participants |
| Placebo BID | Number of Participants With Treatment Emergent Adverse Events. | 14 Participants |
Secondary
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.
Time frame: Day 84
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| JBT-101 20 mg QD | JBT-101 (Lenabasum) Plasma Concentrations on Day 84 | 249.77 ng/mL | Standard Deviation 339.938 |
| JBT-101 20 mg BID | JBT-101 (Lenabasum) Plasma Concentrations on Day 84 | 360.80 ng/mL | Standard Deviation 352.772 |
| Placebo BID | JBT-101 (Lenabasum) Plasma Concentrations on Day 84 | 0.00 ng/mL | Standard Deviation 0 |