Diffuse Cutaneous Systemic Sclerosis
Conditions
Keywords
Scleroderma, cannabinoid receptor type 2 agonist, anabasum, JBT-101, lenabasum
Brief summary
This is a Phase 3 multicenter, double-blind, randomized, placebo-controlled study assessing the efficacy and safety of lenabasum for the treatment of diffuse cutaneous systemic sclerosis (SSc). Approximately 354 subjects will be enrolled in this study at about 60 sites in North America, Europe, Australia, and Asia. The planned duration of treatment with study drug is 52 weeks.
Interventions
DRUGLenabasum 5 mg
Subjects will receive lenabasum 5 mg twice daily.
DRUGLenabasum 20 mg
Subjects will receive lenabasum 20 mg twice daily.
OTHERPlacebo oral capsule
Subjects will receive placebo twice daily.
Sponsors
Corbus Pharmaceuticals Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: 1. ≥ 18 years of age at the time Informed Consent is signed. 2. Diffuse cutaneous SSc (skin thickening on upper arms, upper legs, or trunk). 3. Disease duration ≤ 6 years from the first non-Raynaud's symptom. 4. No new or increased doses of immunosuppressive medications within 8 weeks prior to Screening. Key
Exclusion criteria
1. Unstable SSc or SSc with end-stage organ involvement at Screening or Visit 1. 2. Any of the following values for laboratory tests at Screening: 1. A positive pregnancy test in women of childbearing potential; 2. Hemoglobin \< 9 g/dL for males and \< 8 g/dL for females; 3. Neutrophils \< 1.0 ×10\^9/L; 4. Platelets \< 75 ×10\^9/L; 5. Creatinine clearance \< 50 mL/min according to the Modification of Diet in Renal Disease (MDRD) Study equation; 6. Aspartate aminotransferase or alanine aminotransferase \> 2.0 × upper limit of normal. 3. Any medical condition or concurrent medical therapies at Screening or Visit 1, including a history of non-compliance with medical treatments, that may put the subject at greater safety risk, influence response to study product, or interfere with study assessments.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Efficacy of lenabasum compared to placebo for the American College of Rheumatology Combined Response Index in diffuse cutaneous Systemic Sclerosis score. | American College of Rheumatology Combined Response Index score through study completion, up to 1 year. | The ACR CRISS exponential algorithm determines the predicted probability of improvement from baseline, incorporating change in mRSS, FVC % predicted, physician and patient global assessments, and HAQ-DI. The outcome is a continuous variable between 0.0 and 1.0 (0 - 100%). A higher score indicates greater improvement. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Efficacy of lenabasum compared to placebo for the change from baseline in modified Rodnan skin score. | Change from baseline through study completion, up to 1 year. | mRSS evaluates a subject's skin thickness on a 4 point scale for 17 surface anatomic areas: 0 = normal skin; 1 = mild thickness; 2 = moderate thickness; 3 = severe thickness with inability to pinch skin into fold. The individual values of the 17 surface areas are summed to define the total skin score with a maximum score of 51. |
| Efficacy of lenabasum compared to placebo for the change from baseline in Health Assessment Questionnaire - Disability Index. | Change from baseline through study completion, up to 1 year. | It includes 8 sections: dressing, arising, eating, walking, hygiene, reach, grip, and activities. There are 2 or 3 questions for each section. Scoring within each section is from 0 (without any difficulty) to 3 (unable to do). The individual scores of the eight sections are summed and divided by 8. The result is the disability index or functional disability index. A higher score indicates more functional disability. |
| Efficacy of lenabasum compared to placebo for the change from baseline in forced vital capacity. | Change from baseline through study completion, up to 1 year. | — |
Countries
Australia, Canada, Germany, Israel, Japan, Netherlands, Poland, South Korea, Spain, Switzerland, United Kingdom, United States
Outcome results
None listed