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

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

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.

Study Overview

• Status: Terminated
• Conditions: Dermatomyositis
• Intervention / Treatment: Drug: JBT-101; Drug: Placebo

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Actual): 22
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • University of Pennsylvania Perlman School of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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:

  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. Aspartate aminotransferase, alanine aminotransferase, or alkaline phosphatase > 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: JBT-101; 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; 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.; Other Names: lenabasum
Placebo Comparator: Placebo; 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; 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.

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Corbus Pharmaceuticals Inc.
• Collaborators: University of Pennsylvania; National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
• Investigators: Principal Investigator: Victoria Werth, M.D., University of Pennsylvania Perlman School of Medicine

Publications and helpful links

General Publications

• Werth VP, Hejazi E, Pena SM, Haber J, Zeidi M, Reddy N, Okawa J, Feng R, Bashir MM, Gebre K, Jadoo AS, Concha JSS, Dgetluck N, Constantine S, White B. Safety and Efficacy of Lenabasum, a Cannabinoid Receptor Type 2 Agonist, in Patients with Dermatomyositis with Refractory Skin Disease: A Randomized Clinical Trial. J Invest Dermatol. 2022 Oct;142(10):2651-2659.e1. doi: 10.1016/j.jid.2022.03.029. Epub 2022 Apr 29.

Study record dates

Study Major Dates

• Study Start: June 1, 2015
• Primary Completion (Actual): August 1, 2017
• Study Completion (Actual): January 29, 2021

Study Registration Dates

• First Submitted: June 2, 2015
• First Submitted That Met QC Criteria: June 4, 2015
• First Posted (Estimate): June 9, 2015

Study Record Updates

• Last Update Posted (Estimate): January 19, 2023
• Last Update Submitted That Met QC Criteria: December 23, 2022
• Last Verified: December 1, 2022

More Information

Terms related to this study

• Keywords: JBT-101, lenabasum, dermatomyositis
• Additional Relevant MeSH Terms: Nervous System Diseases; Skin Diseases; Musculoskeletal Diseases; Connective Tissue Diseases; Muscular Diseases; Neuromuscular Diseases; Polymyositis; Myositis; Dermatomyositis; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Cannabinoid Receptor Agonists; Cannabinoid Receptor Modulators; Lenabasum
• Other Study ID Numbers: JBT101-DM-001; 116313 (Investigation New Drug Application (IND)); R21AR066286 (U.S. NIH Grant/Contract)