Safety, Tolerability, Efficacy, and Pharmacokinetics of JBT-101 in Systemic Sclerosis

Corbus Pharmaceuticals

Status and phase: Terminated; Phase 2

Conditions: Diffuse Cutaneous Systemic Sclerosis

Treatments: Drug: Placebo; Drug: JBT-101; Drug: Part B Open-Label Extension

Study type: Interventional

Funder types: Industry

Identifiers

NCT02465437

JBT101-SSc-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 diffuse cutaneous systemic sclerosis.

Full description

Part A of the study is an interventional, double-blind, randomized, placebo-control design will be used to test safety, tolerability, pharmacokinetics, and efficacy of JBT-101 in subjects ≥ 18 and ≤ 70 years of age with active diffuse cutaneous systemic sclerosis. The screening period is up to 28 days, with 84 days treatment period and 28 days follow-up off active treatment.

Part B of the study is an interventional, open-label design will be used. All subjects who complete dosing in Part A without permanent discontinuation of study drug and who pass repeat safety screening will be eligible for enrollment. The screening period is up to 28 days, with a 364 day treatment period and 28 day follow up after last dose of JBT-101.

Enrollment: 42 patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria:

Part A

• Diffuse cutaneous systemic sclerosis
• Have skin thickening from SSc in a body area suitable for repeat biopsy
• Disease duration ≤ 3 years from the first non-Raynaud's phenomenon or >3 years and ≤ 6 years from the first non-Raynaud's phenomenon and high sensitivity C-reactive protein > 3 mg/L, high sensitivity interleukin-6 > 5 pg/mL, or increase in mRSS ≥ 5 points over the last 6 months with total RSS ≥ 12.
• Stable treatment for SSc for at least 28 days before Visit 1

Part B

•Completion of dosing in Part A without permanent discontinuation of study product because of safety or tolerability reasons.

Exclusion Criteria (Part A and B):

• Severe or unstable systemic sclerosis

• Significant diseases or conditions other than systemic sclerosis that may influence response to the study product or safety;

• 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 > 2.0 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: Parallel Assignment

Masking: Double Blind

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42 participants in 5 patient groups, including a placebo group

JBT-101 5 mg/20 mg bid

Experimental group

Description:

JBT-101 5 mg q am and placebo q pm on Days 1-28, then JBT-101 20 mg twice a day (bid) on Days 29-84.

Treatment:

Drug: JBT-101

Drug: Placebo

JBT-101 20 mg/20 mg bid

Experimental group

Description:

JBT-101 20 mg q am and placebo q pm on Days 1-28, then JBT-101 20 mg bid on Days 29-84.

Treatment:

Drug: JBT-101

Drug: Placebo

JBT-101 20 mg bid/20 mg bid

Experimental group

Description:

JBT-101 20 mg bid on Days 1-84.

Treatment:

Drug: JBT-101

Placebo

Placebo Comparator group

Description:

Placebo bid on Days 1-84.

Treatment:

Drug: Placebo

Part B Open-label

Experimental group

Description:

JBT-101 20 mg bid on Days 1-364

Treatment:

Drug: Part B Open-Label Extension