Safety, Tolerability, and Efficacy of JBT-101 in Subjects With Dermatomyositis

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the safety, tolerability and efficacy of JBT-101 in adult subjects with skin-predominant, dermatomyositis (DM) that is refractory to at least 3 months treatment with hydroxychloroquine.

Detailed Description
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Part A: An interventional, double-blind, randomized, placebo-control design will be used to test JBT-101 in about 22 eligible male or female subjects ≥ 18 and ≤ 70 years of age with moderate-to-severe active skin-predominant dermatomyositis. Part B: A one-year open-label design to test JBT-101 in subjects who completed Part A without permanent discontinuation of study product because of safety or tolerability reasons.

Official Title
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A Phase 2, Double-blind, Randomized, Placebo-controlled Study to Investigate the Safety, Tolerability, and Efficacy of JBT-101 in Subjects With Dermatomyositis

Conditions
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Dermatomyositis

Intervention / Treatment
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* Drug: JBT-101
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria (Part A): CDASI activity score ≥ 14; No difficulty with lifting or walking, and no more than 1.5 x the upper limit of normal of creatine phosphokinase or aldolase; Failed at least 3 months treatment with hydroxychloroquine; Stable treatment for dermatomyositis for at least 28 days before Visit 1 (Day 1). Inclusion Criteria (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): Significant diseases or conditions other than DM that may influence response to the study product or safety; Any one of the following values for laboratory tests at Screening: A positive pregnancy test (or at Visit 1); Hemoglobin < 10 g/dL; Neutrophils < 1.0 x 10^9/L; Platelets < 75 x 10^9/L; Creatinine clearance < 50 ml/min according to modified Cockcroft-Gault equation; Aspartate aminotransferase, alanine aminotransferase, or alkaline phosphatase > 2.5 x upper normal limit; 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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: JBT-101<br>Part A: JBT-101 20 mg capsule once a day on Days 1-28, then 20 mg capsule twice a day on Days 29-84. Part B: JBT-101 20 mg twice daily on Days 1 - 365 of the OLE. | Drug: JBT-101<br>* Part A: 20 mg once daily on Days 1-28, then 20 mg twice daily on Days 29-84. Part B: JBT-101 20 mg twice daily on Days 1 - 365 of the OLE.<br>* Other names: lenabasum;|
| Placebo Comparator: Placebo<br>Part A: Placebo capsule once a day on Days 1-28, then placebo capsule twice a day on Days 29-84. Part B: Placebo twice daily on Days 1 - 365 of the OLE. | Drug: Placebo<br>* Part A: Once daily on Days 1-28, then twice daily on Days 29-84. Part B: Placebo twice daily on Days 1 - 365 of the OLE.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) From Baseline in Part A. | The CDASI is a validated outcome measure that systematically quantifies cutaneous DM disease activity, In the CDASI, DM skin disease activity is scored from 0 to 100 based on the physician's evaluation of erythema, scale, and erosion or ulceration at 15 anatomic locations as well as alopecia, Gottron's sign or papules on the hands, and periungual changes. A 5-point or greater decrease in the CDASI activity score indicates clinically relevant improvement based on statistical analysis using a receiver operating characteristic curve to maximize sensitivity and specificity | Part A: 84-day treatment period (Change from the Baseline CDSAI score at Day 84) | 
| Number of Participants With Treatment Emergent Adverse Events as a Measure of Safety and Tolerability | Number of participants with treatment emergent adverse events were assessed as a measure of safety and tolerability | Part A: to Day 84 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Patient-reported Outcomes From Baseline at 84 Days for Part A | LS mean (SE) change from baseline to Week 6 (Day 84) for lenabasum vs. placebo using a mixed model repeated measures analysis The CDASI is a validated outcome measure that systematically quantifies cutaneous DM disease activity and damage, In the CDASI, the Damage Score is scored from 0 to 32 based on the physician's evaluation of poikiloderma and calcinosis. 0 representing no damage and 32 representing the greatest level of damage. | Part A: 84-day treatment period | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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JBT-101, lenabasum, dermatomyositis