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

Corbus Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Cystic Fibrosis

Treatments: Drug: JBT-101 (lenabasum); Other: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02465450

JBT101-CF-001

Details and patient eligibility

About

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

Full 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.

Enrollment: 85 patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Double Blind

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85 participants in 6 patient groups, including a placebo group

JBT101 (lenabasum) 1 mg

Experimental group

Description:

JBT-101 1 mg once a day on Days 1-28

Treatment:

Drug: JBT-101 (lenabasum)

JBT-101 (lenabasum) 5 mg

Experimental group

Description:

JBT-101 5 mg once a day on Days 1-28

Treatment:

Drug: JBT-101 (lenabasum)

Placebo

Placebo Comparator group

Description:

Placebo once a day on Days 1-28.

Treatment:

Other: Placebo

JBT-101 (lenabasum) 20 mg QD

Experimental group

Description:

JBT-101 20 mg once a day on Days 29-84.

Treatment:

Drug: JBT-101 (lenabasum)

JBT-101 (lenabasum) 20 mg BID

Experimental group

Description:

JBT-101 20 mg twice daily on Days 29-84.

Treatment:

Drug: JBT-101 (lenabasum)

Placebo BID

Placebo Comparator group

Description:

Placebo twice daily on Days 29-84.

Treatment:

Other: Placebo