Clinical trial NCT02465437: Safety, Tolerability, Efficacy, and Pharmacokinetics of JBT-101 in Systemic Sclerosis
Official title: A Phase 2, Double-blind, Randomized, Placebo-controlled Multicenter Study to Evaluate Safety, Tolerability, Efficacy, and Pharmacokinetics of JBT-101 in Diffuse Cutaneous Systemic Sclerosis
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Diffuse Cutaneous Systemic Sclerosis
Keywords: JBT-101, Lenabasum, Systemic Sclerosis
Lead sponsor: Corbus Pharmaceuticals Inc.
Sponsor class: INDUSTRY
First posted: 2015-06-08
Enrollment: 42
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.

Detailed 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.

Eligibility 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.

Primary outcomes:
measure: Number of Participants With Treatment-emergent Adverse Events From Baseline at Day 113
timeFrame: Part A: Day 113
measure: Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (CRISS) at Day 85 and 113
timeFrame: Day 85 and Day 113
Secondary outcomes:
measure: CRISS Individual Components (mRSS Total Score) Change From Baseline.
timeFrame: Day 85 and 113
measure: CRISS Individual Component (FVC Percent Predicted) Change From Baseline
timeFrame: Day 85 and 113
measure: CRISS Individual Component (Physician Global Assessment Score) Change From Baseline
timeFrame: Day 85 and 113
measure: CRISS Individual Component (Patient Global Assessment Score) Change From Baseline
timeFrame: Day 85 and 113
measure: CRISS Individual Component (HAQ-DI Score) Change From Baseline.
timeFrame: Day 85 and 113
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Arthritis Association of Southern CA
state: California
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: Baltimore
country: United States
facility: John Hopkins Scleroderma Center
state: Maryland
city: Boston
country: United States
facility: Boston University Medical Center
state: Massachusetts
city: New Brunswick
country: United States
facility: Rutgers University
state: New Jersey
city: New York
country: United States
facility: Weill Cornell Medical College
state: New York
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center
state: Pennsylvania
city: Houston
country: United States
facility: University of Texas Houston Medical School
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
Study officials:
affiliation: Weill Cornell Medical College, New York City, NY
name: Robert Spiera, M.D.
role: PRINCIPAL_INVESTIGATOR
References: